[et_pb_section fb_built=\u201d1\u2033 fullwidth=\u201don\u201d disabled_on=\u201doff|off|off\u201d _builder_version=\u201d4.16\u2033 border_width_bottom=\u201d55px\u201d border_color_bottom=\u201d#29327a\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55px\u201d background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/09\/Kids-on-Bridge.png\u201d custom_padding=\u201d11.5vw||11.5vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited=\u201dsur|bureau\u201d title_font_size_tablet=\u201d45px\u201d title_font_size_phone=\u201d40px\u201d title_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 custom_css_main_element=\u201dbackground-position: center 18% !important;\u201d title_text_shadow_horizontal_length_tablet=\u201d0px\u201d title_text_shadow_vertical_length_tablet=\u201d0px\u201d title_text_shadow_blur_strength_tablet=\u201d1px\u201d content_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_text_shadow_vertical_length_tablet=\u201d0px\u201d content_text_shadow_blur_strength_tablet=\u201d1px\u201d subhead_text_shadow_horizontal_length_tablet=\u201d0px\u201d subhead_text_shadow_vertical_length_tablet=\u201d0px\u201d subhead_text_shadow_blur_strength_tablet=\u201d1px\u201d content_link_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_link_text_shadow_vertical_length_tablet=\u201d0px\u201d content_link_text_shadow_blur_strength_tablet=\u201d1px\u201d content_ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_ul_text_shadow_vertical_length_tablet=\u201d0px\u201d content_ul_text_shadow_blur_strength_tablet=\u201d1px\u201d content_ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_ol_text_shadow_vertical_length_tablet=\u201d0px\u201d content_ol_text_shadow_blur_strength_tablet=\u201d1px\u201d content_quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_quote_text_shadow_vertical_length_tablet=\u201d0px\u201d content_quote_text_shadow_blur_strength_tablet=\u201d1px\u201d button_one_text_shadow_horizontal_length_tablet=\u201d0px\u201d button_one_text_shadow_vertical_length_tablet=\u201d0px\u201d button_one_text_shadow_blur_strength_tablet=\u201d1px\u201d button_two_text_shadow_horizontal_length_tablet=\u201d0px\u201d button_two_text_shadow_vertical_length_tablet=\u201d0px\u201d button_two_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d box_shadow_horizontal_image_tablet=\u201d0px\u201d box_shadow_vertical_image_tablet=\u201d0px\u201d box_shadow_blur_image_tablet=\u201d40px\u201d box_shadow_spread_image_tablet=\u201d0px\u201d box_shadow_horizontal_button_one_tablet=\u201d0px\u201d box_shadow_vertical_button_one_tablet=\u201d0px\u201d box_shadow_blur_button_one_tablet=\u201d40px\u201d box_shadow_spread_button_one_tablet=\u201d0px\u201d box_shadow_horizontal_button_two_tablet=\u201d0px\u201d box_shadow_vertical_button_two_tablet=\u201d0px\u201d box_shadow_blur_button_two_tablet=\u201d40px\u201d box_shadow_spread_button_two_tablet=\u201d0px\u201d text_shadow_horizontal_length_tablet=\u201d0px\u201d text_shadow_vertical_length_tablet=\u201d0px\u201d text_shadow_blur_strength_tablet=\u201d1px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n
<\/p>\n
[\/et_pb_fullwidth_header][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 specialty=\u201don\u201d padding_left_1=\u201d35px\u201d padding_left_2=\u201d35px\u201d padding_2_tablet=\u201d|||0px\u201d padding_2_phone=\u201d|||0px\u201d padding_2_last_edited=\u201don|desktop\u201d module_class_1=\u201dsidebar-secondary-nav\u201d module_class=\u201dempreinte-de-la-main-bg\u201d _builder_version=\u201d4.16\u2033 background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/empreinte-de-la-main-bleue-uniquement.png\u201d parallax=\u201don\u201d parallax_method=\u201doff\u201d inner_width=\u201d100%\u201d inner_max_width=\u201d100%\u201d custom_padding=\u201d0|0px|54px|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d10px\u201d border_color_top=\u201d#8fd2ed\u201d use_custom_width=\u201don\u201d width_unit=\u201doff\u201d custom_width_percent=\u201d100%\u201d global_colors_info=\u201d{}\u201d][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_sidebar area=\u201det_pb_widget_area_1\u2033 disabled_on=\u201don|on|off\u201d module_class=\u201dsubpage-sidebars\u201d _builder_version=\u201d4.16\u2033 animation_style=\u201dfade\u201d z_index_tablet=\u201d500\u2033 border_width_right=\u201d5px\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d]
\n[\/et_pb_sidebar][\/et_pb_column][et_pb_column type=\u201d3_4\u2033 specialty_columns=\u201d3\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|phone\u201d disabled_on=\u201don|on|\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d|35px||0px\u201d custom_padding_tablet=\u201d|35px||35px||true\u201d custom_padding_phone=\u201d\u201d animation_direction=\u201dtop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dEssais cliniques du m\u00e9dicament Progeria : Contexte\u201d _builder_version=\u201d4.27.2\u2033 header_4_line_height=\u201d1.2em\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d header_text_shadow_blur_strength_tablet=\u201d1px\u201d header_2_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_2_text_shadow_vertical_length_tablet=\u201d0px\u201d header_2_text_shadow_blur_strength_tablet=\u201d1px\u201d header_3_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_3_text_shadow_vertical_length_tablet=\u201d0px\u201d header_3_text_shadow_blur_strength_tablet=\u201d1px\u201d header_4_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_4_text_shadow_vertical_length_tablet=\u201d0px\u201d header_4_text_shadow_blur_strength_tablet=\u201d1px\u201d header_5_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_5_text_shadow_vertical_length_tablet=\u201d0px\u201d header_5_text_shadow_blur_strength_tablet=\u201d1px\u201d header_6_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_6_text_shadow_vertical_length_tablet=\u201d0px\u201d header_6_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/HayleyMichielAmber-Aug09.jpg\")
Les essais cliniques sur les m\u00e9dicaments contre la prog\u00e9ria repr\u00e9sentent le meilleur espoir pour les enfants atteints de cette maladie. Ils permettent de tester des traitements potentiels qui pourraient leur permettre de vivre plus longtemps et en meilleure sant\u00e9. Ces essais sont l'aboutissement d'ann\u00e9es de recherche ax\u00e9es sur le m\u00e9dicament ou la combinaison de m\u00e9dicaments qui traiteront et gu\u00e9riront les enfants.<\/p>\n
Depuis la cr\u00e9ation de la PRF en 1999, alors qu'il n'existait aucune ressource pour ces enfants, nous sommes pass\u00e9s d'une obscurit\u00e9 totale \u00e0 la d\u00e9couverte de g\u00e8nes, aux premiers essais cliniques sur la prog\u00e9ria, au tout premier traitement approuv\u00e9 par la FDA, appel\u00e9 lonafarnib, le tout \u00e0 un rythme pratiquement in\u00e9dit dans la communaut\u00e9 scientifique. Et tout en aidant cette poign\u00e9e d'enfants, le lien entre la prog\u00e9ria et les maladies cardiaques courantes et le vieillissement a d'\u00e9normes implications pour nous tous.<\/p>\n
[\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dQuelle est la prochaine \u00e9tape pour les traitements et la gu\u00e9rison ? \u201d _builder_version=\u201d4.27.2\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d custom_margin=\u201d|||15px|false|false\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n
<\/p>\n
[\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 rembourrage_personnalis\u00e9=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dD\u00e9marrage d\u2019un nouvel essai clinique de m\u00e9dicament\u201d _builder_version=\u201d4.27.2\u2033 header_4_line_height=\u201d1.2em\u201d vertical_offset_tablet=\u201d0\u2033 horizontal_offset_tablet=\u201d0\u2033 custom_margin=\u201d||25px\u201d hover_enabled=\u201d0\u2033 header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d header_text_shadow_blur_strength_tablet=\u201d1px\u201d header_2_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_2_text_shadow_vertical_length_tablet=\u201d0px\u201d header_2_text_shadow_blur_strength_tablet=\u201d1px\u201d header_3_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_3_text_shadow_vertical_length_tablet=\u201d0px\u201d header_3_text_shadow_blur_strength_tablet=\u201d1px\u201d header_4_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_4_text_shadow_vertical_length_tablet=\u201d0px\u201d header_4_text_shadow_blur_strength_tablet=\u201d1px\u201d header_5_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_5_text_shadow_vertical_length_tablet=\u201d0px\u201d header_5_text_shadow_blur_strength_tablet=\u201d1px\u201d header_6_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_6_text_shadow_vertical_length_tablet=\u201d0px\u201d header_6_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d sticky_enabled=\u201d0\u2033]<\/p>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/11\/Bastianforweb-449x1024.png\")
<\/strong><\/em><\/strong><\/h4>\n<\/em><\/strong><\/p>\n PRF, en collaboration avec le sponsor de l'\u00e9tude bas\u00e9 en Cor\u00e9e PRG Science & Technology (PRG S&T), a commenc\u00e9 le recrutement pour un tout nouvel essai clinique avec un m\u00e9dicament appel\u00e9 Progerinin. Des preuves de laboratoire montrent que ce m\u00e9dicament, lorsqu'il est pris en association avec le lonafarnib, peut \u00eatre plus efficace que le lonafarnib seul. PRF a financ\u00e9 les travaux de laboratoire qui ont conduit \u00e0 la formation de PRG S&T et au d\u00e9veloppement de Progerinin. Les travaux pr\u00e9liminaires \u00e0 l'essai ont commenc\u00e9 au Boston Children's Hospital, en pr\u00e9vision de l'inscription d'enfants du monde entier \u00e0 cet essai dans les mois \u00e0 venir. Nous sommes tr\u00e8s enthousiastes \u00e0 l'id\u00e9e de lancer un nouvel essai avec un m\u00e9dicament aussi prometteur et avons h\u00e2te de partager plus de d\u00e9tails avec vous d\u00e8s qu'ils seront disponibles.<\/p>\n Veuillez contacter Shelby Phillips pour en savoir plus sur le proc\u00e8s. La cofondatrice et directrice ex\u00e9cutive du PRF, Audrey Gordon, conclut en juin un accord avec le Dr Bum-Joon Park de PRG S&T pour avancer dans les travaux pr\u00e9liminaires au proc\u00e8s.<\/em><\/p>\n <\/em><\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dTh\u00e9rapie par ARN : l\u2019\u00e9tude de faisabilit\u00e9 de l\u2019administration du m\u00e9dicament est termin\u00e9e !\u201d _builder_version=\u201d4.27.2\u2033 header_4_line_height=\u201d1.2em\u201d vertical_offset_tablet=\u201d0\u2033 horizontal_offset_tablet=\u201d0\u2033 custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d header_text_shadow_blur_strength_tablet=\u201d1px\u201d header_2_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_2_text_shadow_vertical_length_tablet=\u201d0px\u201d header_2_text_shadow_blur_strength_tablet=\u201d1px\u201d header_3_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_3_text_shadow_vertical_length_tablet=\u201d0px\u201d header_3_text_shadow_blur_strength_tablet=\u201d1px\u201d header_4_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_4_text_shadow_vertical_length_tablet=\u201d0px\u201d header_4_text_shadow_blur_strength_tablet=\u201d1px\u201d header_5_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_5_text_shadow_vertical_length_tablet=\u201d0px\u201d header_5_text_shadow_blur_strength_tablet=\u201d1px\u201d header_6_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_6_text_shadow_vertical_length_tablet=\u201d0px\u201d header_6_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n Le PRF a fait les premiers pas avec les patients vers un essai clinique sur la th\u00e9rapie par ARN \u2013 tellement excitant !<\/p>\n Contexte : En janvier 2021, nous avons signal\u00e9 d\u00e9couvertes r\u00e9volutionnaires dans le domaine th\u00e9rapeutique de l'ARN<\/strong>, dans lequel cette th\u00e9rapie production inhib\u00e9e d'ARN codant pour la prot\u00e9ine responsable de la maladie de Progeria, la prog\u00e9rine<\/strong>. L'\u00e9tude*, dirig\u00e9e par le Dr Francis Collins, conseiller scientifique de la Maison Blanche et ancien directeur des National Institutes of Health (NIH), a r\u00e9v\u00e9l\u00e9 que des souris atteintes de Progeria trait\u00e9es avec un m\u00e9dicament appel\u00e9 SRP-2001 r\u00e9duit l'expression de l'ARNm et des prot\u00e9ines nocives de la prog\u00e9rine dans les vaisseaux sanguins<\/strong>, ainsi que dans d'autres tissus. Les vaisseaux sanguins \u00e9taient plus forts et les souris ont montr\u00e9 une augmentation de la survie de plus de 60%<\/strong> par rapport aux souris non trait\u00e9es. Ainsi, le travail avec cette th\u00e9rapie prometteuse s'est poursuivi et nous avons pris la\u00a0prochaine \u00e9tape <\/strong>avec un \u00c9tude de faisabilit\u00e9 <\/strong>comme suit:<\/p>\n En r\u00e8gle g\u00e9n\u00e9rale, les th\u00e9rapies \u00e0 base d'ARN sont des liquides inject\u00e9s par voie intraveineuse (directement dans la veine). Cependant, les personnes atteintes de prog\u00e9ria ne seraient pas en mesure de tol\u00e9rer l'administration intraveineuse de la dose quotidienne requise. Ainsi, PRF a d\u00e9velopp\u00e9 une syst\u00e8me d'administration sous-cutan\u00e9e<\/strong> gr\u00e2ce \u00e0 quoi le liquide peut \u00eatre inject\u00e9 avec une petite aiguille sous la peau. Une \u00e9tude de 6 mois a \u00e9t\u00e9 r\u00e9alis\u00e9e au BCH pour d\u00e9terminer la faisabilit\u00e9 de cette approche d'administration pour les personnes atteintes de prog\u00e9ria<\/strong>L'\u00e9quipe a test\u00e9 si l'administration d'une solution saline pouvait \u00eatre confortablement inject\u00e9e par voie sous-cutan\u00e9e et quel syst\u00e8me et site d'injection seraient les plus confortables. Une fois les donn\u00e9es de cette \u00e9tude analys\u00e9es, nous serons Un pas de plus vers un essai clinique en th\u00e9rapie g\u00e9nique<\/strong>!<\/p>\n *<\/strong>Erdos, MR, Cabral, WA, Tavarez, UL et al. Une approche th\u00e9rapeutique antisens cibl\u00e9e pour le syndrome de prog\u00e9ria de Hutchinson-Gilford. M\u00e9decine Nat<\/em> (2021).<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dQuelle est la prochaine \u00e9tape pour les traitements et la gu\u00e9rison ? \u201d _builder_version=\u201d4.27.0\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d custom_margin=\u201d|||15px|false|false\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dQue se passe-t-il AUJOURD\u2019HUI avec les essais cliniques sur la prog\u00e9ria ? \u201d _builder_version=\u201d4.27.0\u2033 header_4_line_height=\u201d1.2em\u201d vertical_offset_tablet=\u201d0\u2033 horizontal_offset_tablet=\u201d0\u2033 custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 text_text_shadow_horizontal_length_tablet=\u201d0px\u201d text_text_shadow_vertical_length_tablet=\u201d0px\u201d text_text_shadow_blur_strength_tablet=\u201d1px\u201d link_text_shadow_horizontal_length_tablet=\u201d0px\u201d link_text_shadow_vertical_length_tablet=\u201d0px\u201d link_text_shadow_blur_strength_tablet=\u201d1px\u201d ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d ul_text_shadow_vertical_length_tablet=\u201d0px\u201d ul_text_shadow_blur_strength_tablet=\u201d1px\u201d ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d ol_text_shadow_vertical_length_tablet=\u201d0px\u201d ol_text_shadow_blur_strength_tablet=\u201d1px\u201d quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d quote_text_shadow_vertical_length_tablet=\u201d0px\u201d quote_text_shadow_blur_strength_tablet=\u201d1px\u201d header_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_text_shadow_vertical_length_tablet=\u201d0px\u201d header_text_shadow_blur_strength_tablet=\u201d1px\u201d header_2_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_2_text_shadow_vertical_length_tablet=\u201d0px\u201d header_2_text_shadow_blur_strength_tablet=\u201d1px\u201d header_3_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_3_text_shadow_vertical_length_tablet=\u201d0px\u201d header_3_text_shadow_blur_strength_tablet=\u201d1px\u201d header_4_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_4_text_shadow_vertical_length_tablet=\u201d0px\u201d header_4_text_shadow_blur_strength_tablet=\u201d1px\u201d header_5_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_5_text_shadow_vertical_length_tablet=\u201d0px\u201d header_5_text_shadow_blur_strength_tablet=\u201d1px\u201d header_6_text_shadow_horizontal_length_tablet=\u201d0px\u201d header_6_text_shadow_vertical_length_tablet=\u201d0px\u201d header_6_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n L'essai le plus r\u00e9cent portait sur 2 m\u00e9dicaments : lonafarnib<\/strong>\u00a0et un nouveau m\u00e9dicament, \u00e9v\u00e9rolimus<\/strong>La phase 1, visant \u00e0 d\u00e9terminer le dosage s\u00fbr et appropri\u00e9 de l'\u00e9v\u00e9rolimus, a d\u00e9but\u00e9 en avril 2016 et s'est achev\u00e9e avec succ\u00e8s en juin 2017. La phase 2, qui a test\u00e9 l'efficacit\u00e9 de l'association de deux m\u00e9dicaments, a d\u00e9but\u00e9 en juillet 2017 et s'est achev\u00e9e en avril 2022. Soixante enfants de 27 pays ont \u00e9t\u00e9 inclus dans cette phase \u00e0 deux m\u00e9dicaments !<\/p>\n L\u2019\u00e9v\u00e9rolimus est une forme de rapamycine, un m\u00e9dicament qui pourrait \u00eatre administr\u00e9 plus facilement aux enfants atteints de prog\u00e9ria, car il n\u00e9cessite moins de prises de sang pour mesurer les concentrations du m\u00e9dicament. Alors que le lonafarnib peut bloquer le d\u00e9veloppement de la prog\u00e9rine toxique, la rapamycine semble permettre aux cellules d\u2019\u00e9liminer plus rapidement la prog\u00e9rine. Ainsi, la rapamycine ciblant une voie diff\u00e9rente de celle du lonafarnib, la combinaison pourrait s\u2019av\u00e9rer \u00eatre un \u00ab coup double \u00bb contre la prog\u00e9ria \u2013 et, esp\u00e9rons-le, un meilleur traitement que le lonafarnib seul.<\/p>\n Nous sommes d\u00e9sormais entr\u00e9s dans une p\u00e9riode d\u2019analyse des donn\u00e9es et nous pr\u00e9voyons de publier les r\u00e9sultats dans une revue scientifique \u00e0 comit\u00e9 de lecture. Entre-temps, les participants \u00e0 l\u2019essai ont soit opt\u00e9 pour l\u2019extension de l\u2019essai en monoth\u00e9rapie, soit pour un autre programme d\u2019acc\u00e8s. Quelle que soit la voie choisie, les participants continuent de recevoir du lonafarnib, le traitement de r\u00e9f\u00e9rence actuel.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dHistorique des essais en un coup d\u2019\u0153il\u201d _builder_version=\u201d4.27.0\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d custom_margin=\u201d||15px|false|false\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|phone\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.22.1\u2033 custom_margin=\u201d0px|||false|false\u201d custom_padding=\u201d|35px|0px|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201d\u00c0 ce jour, le PRF a financ\u00e9 et coordonn\u00e9 trois essais cliniques\u201d _builder_version=\u201d4.27.0\u2033 text_font=\u201d||||||||\u201d header_4_font=\u201d||||||||\u201d header_4_line_height=\u201d1.2em\u201d custom_padding=\u201d||0px|||\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d global_colors_info=\u201d{}\u201d]<\/p>\n \u00c0 ce jour, le PRF a financ\u00e9 et coordonn\u00e9 quatre essais cliniques<\/strong>. La PRF est et a toujours \u00e9t\u00e9 responsable de tous les frais des essais, y compris les tests, les d\u00e9placements, la nourriture, l'h\u00e9bergement, les traducteurs et le personnel. Chaque nouvel essai est plus co\u00fbteux que le pr\u00e9c\u00e9dent, car de plus en plus d'enfants s'inscrivent pour avoir une chance de vivre plus longtemps et en meilleure sant\u00e9.<\/p>\n [\/et_pb_text][et_pb_button button_url=\u201dhttps:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\u201d url_new_window=\u201don\u201d button_text=\u201dAidez-nous \u00e0 financer ces essais vitaux. Faites un don aujourd\u2019hui\u201d admin_label=\u201dFaire un don\u201d _builder_version=\u201d4.22.1\u2033 background_layout=\u201ddark\u201d custom_margin=\u201d20px||25px\u201d animation_style=\u201dslide\u201d animation_direction=\u201dleft\u201d animation_intensity_slide=\u201d25%\u201d global_colors_info=\u201d{}\u201d] Le projet #1, qui impliquait un seul m\u00e9dicament, le lonafarnib, a d\u00e9but\u00e9 en 2007 et s'est av\u00e9r\u00e9 efficace. D\u00e9couvrez tout sur cette d\u00e9couverte historique du traitement ici.<\/a><\/p>\n #2, la partie \u00ab faisabilit\u00e9 \u00bb de l\u2019\u00ab essai triple \u00bb impliquait 3 m\u00e9dicaments : le lonafarnib, la pravastatine et le zol\u00e9dronate. Ce \u00ab mini-essai \u00bb de phase 1 d\u2019un mois a eu lieu en mars 2009 pour d\u00e9terminer si l\u2019ajout de 2 m\u00e9dicaments suppl\u00e9mentaires au traitement par le lonafarnib \u00e9tait sans danger pour une population plus large (ce qui \u00e9tait le cas).<\/p>\n L\u2019essai #3, le \u00ab Triple Trial \u00bb, \u00e9tait une continuation de l\u2019essai de faisabilit\u00e9 et une \u00e9tude de phase 2 qui a d\u00e9but\u00e9 en ao\u00fbt 2009. Son protocole a chang\u00e9 au cours des cinq ann\u00e9es suivantes, revenant au seul lonafarnib et r\u00e9ouvrant les inscriptions pour que davantage d\u2019enfants puissent participer. Lire la suite ici<\/a>.<\/p>\n L'essai #4 porte sur deux m\u00e9dicaments, le lonafarnib et l'\u00e9v\u00e9rolimus. La phase 1, visant \u00e0 d\u00e9terminer le dosage s\u00fbr et appropri\u00e9 de l'\u00e9v\u00e9rolimus, a d\u00e9but\u00e9 en avril 2016 et s'est achev\u00e9e avec succ\u00e8s en juin 2017. La phase 2, qui a test\u00e9 l'efficacit\u00e9 de l'association de deux m\u00e9dicaments, a d\u00e9but\u00e9 en juillet 2017 et s'est achev\u00e9e en avril 2022. L'extension de monoth\u00e9rapie de cet essai se poursuit aujourd'hui.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][et_pb_column_inner type=\u201d1_2\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|desktop\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/02\/Ana-Clara-trial-page.jpg\u201d align=\u201dcenter\u201d align_tablet=\u201dcenter\u201d align_phone=\u201d\u201d align_last_edited=\u201don|desktop\u201d admin_label=\u201dliv\u201d _builder_version=\u201d4.22.1\u2033 border_width_all=\u201d4px\u201d border_color_all=\u201d#392887\u2033 global_colors_info=\u201d{}\u201d] En 2006, les chercheurs ont identifi\u00e9 un traitement m\u00e9dicamenteux potentiel pour les enfants atteints de prog\u00e9ria,\u00a0appel\u00e9s FTI.<\/a>\u00a0Pour la premi\u00e8re fois, nous avions devant nous un traitement possible pour les enfants atteints de prog\u00e9ria.\u00a0<\/b>Des moments passionnants ! L'essai clinique du m\u00e9dicament Progeria a d\u00e9but\u00e9 le 7 mai 2007 avec l'arriv\u00e9e de deux enfants \u2013 Meghan et Megan \u2013 au Boston Children's Hospital de Boston, dans le Massachusetts, pour leur premi\u00e8re visite sur sept sur une p\u00e9riode de deux ans. Lors de cette premi\u00e8re visite, elles ont subi des tests approfondis et re\u00e7u leurs premi\u00e8res doses du m\u00e9dicament. En moyenne, deux familles se sont rendues \u00e0 Boston chaque semaine par la suite, jusqu'en d\u00e9cembre 2009, p\u00e9riode pendant laquelle l'\u00e9quipe de l'essai a analys\u00e9 les milliers d'\u00e9l\u00e9ments de donn\u00e9es (chaque enfant a subi plus de 100 tests par visite !) et a demand\u00e9 la publication des r\u00e9sultats.<\/p>\n <\/p>\n [\/et_pb_text][et_pb_testimonial author=\u201dFrancis Collins, MD, PhD\u201d job_title=\u201dDirecteur du National Human Genome Research Institute qui a cartographi\u00e9 le g\u00e9nome humain, conf\u00e9rencier et co-d\u00e9couvreur du g\u00e8ne de la Progeria.\u201d quote_icon_color=\u201d#00b2e2\u2033 portrait_width=\u201d140px\u201d portrait_height=\u201d140px\u201d _builder_version=\u201d4.16\u2033 background_color=\u201d#f7f7f7\u2033 custom_margin_tablet=\u201d\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 custom_css_testimonial_author=\u201dcolor: #00b2e2;||font-size: 19px !important;||font-weight: 700;\u201d custom_css_testimonial_meta=\u201dfont-style: italic;||font-weight: 700;\u201d box_shadow_style_image=\u201dpreset3\u2033 locked=\u201doff\u201d global_colors_info=\u201d{}\u201d]<\/p>\n \u00ab Je ne connais aucune autre maladie g\u00e9n\u00e9tique rare qui soit pass\u00e9e de la d\u00e9couverte du g\u00e8ne \u00e0 l\u2019essai clinique en moins de quatre ans \u2013 un t\u00e9moignage ph\u00e9nom\u00e9nal du travail acharn\u00e9 de la Progeria Research Foundation. \u00bb<\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201d1_2,1_2\u2033 custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dfalse\u201d padding_left_right_link_2=\u201dfalse\u201d _builder_version=\u201d4.16\u2033 custom_margin=\u201d||25px\u201d custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201d1_2\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|20px||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/11\/redoof2007photocollage.png\u201d title_text=\u201dredoof2007photocollage\u201d align_tablet=\u201dcenter\u201d align_phone=\u201d\u201d align_last_edited=\u201dsur|bureau\u201d admin_label=\u201dcollage\u201d _builder_version=\u201d4.27.0\u2033 custom_margin=\u201d|-50px|||false|false\u201d global_colors_info=\u201d{}\u201d] <\/p>\n Vingt-huit (28) enfants de seize pays ont particip\u00e9 \u00e0 l'essai, \u00e2g\u00e9s de 3 \u00e0 15 ans. Les enfants sont retourn\u00e9s \u00e0 l'h\u00f4pital pour enfants de Boston tous les quatre mois pour subir des tests et recevoir de nouveaux m\u00e9dicaments. Ils sont rest\u00e9s \u00e0 Boston pendant 4 \u00e0 8 jours \u00e0 chaque visite. \u00c0 la maison, leurs m\u00e9decins ont surveill\u00e9 de pr\u00e8s les enfants et ont soumis des rapports de sant\u00e9 p\u00e9riodiques \u00e0 l'\u00e9quipe de recherche de Boston. Pendant toute la dur\u00e9e de l'essai, en moyenne 2 enfants par semaine se sont rendus \u00e0 Boston pour participer.<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dQui, o\u00f9, quand, comment et combien\u2026\u201d _builder_version=\u201d4.27.0\u2033 header_text_align=\u201dcenter\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201ddark\u201d animation_style=\u201dfade\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dLes trois premiers essais cliniques ont \u00e9t\u00e9 dirig\u00e9s par le Dr Mark Kieran\u201d _builder_version=\u201d4.27.0\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d custom_margin_tablet=\u201d35px||\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201dsur|bureau\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Les trois premiers essais cliniques ont \u00e9t\u00e9 dirig\u00e9s par le Dr Mark Kieran, PhD,\u00a0<\/strong>Directeur, Neuro-oncologie m\u00e9dicale p\u00e9diatrique, Dana-Farber Cancer Institute et Children's Hospital Boston ; professeur adjoint, d\u00e9partements de p\u00e9diatrie et d'h\u00e9matologie\/oncologie, Harvard Medical School. Le Dr Kieran est un oncologue p\u00e9diatrique poss\u00e9dant une vaste exp\u00e9rience du m\u00e9dicament \u00e0 l'\u00e9tude (farn\u00e9syltransf\u00e9rase, ou FTI) chez les enfants. En 2017, il a quitt\u00e9 son poste chez Dana Farber pour travailler dans le secteur priv\u00e9. Les copr\u00e9sidents \u00e9taient Monica Kleinman, MD, directrice de l'unit\u00e9 de soins intensifs m\u00e9dico-chirurgicaux, associ\u00e9e principale en m\u00e9decine de soins intensifs au BCH, professeur adjoint \u00e0 la Harvard Medical School ; et Leslie Gordon, MD, PhD, directrice m\u00e9dicale du PRF, charg\u00e9e de cours au BCH et \u00e0 la Harvard Medical School, professeur de p\u00e9diatrie au Hasbro Children's Hospital et \u00e0 la Brown University \u00e0 Providence, RI. Le Dr Kleinman a assum\u00e9 le r\u00f4le principal de chercheur principal.<\/p>\n Les essais cliniques sont le fruit d'un effort collaboratif impliquant des m\u00e9decins du Boston Children's Hospital, du Dana-Farber Cancer Institute et du Brigham and Women's Hospital.,<\/strong>\u00a0toutes les institutions de l'Universit\u00e9 Harvard. De plus, des m\u00e9decins et des scientifiques de la Warren Alpert Medical School de l'Universit\u00e9 Brown et du NIH ont contribu\u00e9 au succ\u00e8s de ce premier essai et des suivants.<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dComment en sommes-nous arriv\u00e9s l\u00e0 ?\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n En 2003, l'\u00e9quipe de recherche collaborative de la Fondation de recherche sur la prog\u00e9ria\u00a0\u00a0d\u00e9couvert le g\u00e8ne de la prog\u00e9ria<\/a>.<\/a> Cette d\u00e9couverte a non seulement permis de mieux comprendre la prog\u00e9ria, mais les scientifiques savent d\u00e9sormais que l\u2019\u00e9tude de la prog\u00e9ria peut nous aider \u00e0 en apprendre davantage sur les maladies cardiaques et le processus normal de vieillissement qui nous affecte tous. Depuis la d\u00e9couverte du g\u00e8ne, le soutien des chercheurs, des cliniciens, des familles d\u2019enfants atteints de prog\u00e9ria et des personnes comme VOUS nous a amen\u00e9s \u00e0 un autre carrefour dans la recherche d\u2019un traitement. Les chercheurs ont commenc\u00e9 une \u00e9tude intensive de cette prot\u00e9ine ennemie appel\u00e9e\u00a0prog\u00e9rine<\/em>, et en 2006, ils ont identifi\u00e9 un traitement m\u00e9dicamenteux potentiel pour les enfants atteints de prog\u00e9ria, appel\u00e9 inhibiteurs de la farn\u00e9syltransf\u00e9rase (FTI), et ont men\u00e9 des \u00e9tudes en laboratoire qui ont soutenu un essai sur l'homme avec le m\u00e9dicament. Le FTI choisi a \u00e9t\u00e9 initialement fourni par Merck et appel\u00e9 lonafarnib<\/em>.\u00a0Cliquez ici<\/a>\u00a0pour plus de d\u00e9tails sur la recherche.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dPourquoi les chercheurs pensaient-ils que ce m\u00e9dicament fonctionnerait contre la prog\u00e9ria ?\u201d _builder_version=\u201d4.16\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Cellule normale, cellule Progeria, cellule Progeria apr\u00e8s traitement avec FTI.<\/p><\/div>\n La prot\u00e9ine que nous pensons responsable de la prog\u00e9ria s\u2019appelle la prog\u00e9rine. Pour bloquer la fonction cellulaire normale et provoquer la prog\u00e9ria, une mol\u00e9cule appel\u00e9e \u00ab groupe farn\u00e9syle \u00bb doit \u00eatre attach\u00e9e \u00e0 la prot\u00e9ine prog\u00e9rine. Les FTI agissent en bloquant (inhibant) la fixation du groupe farn\u00e9syle sur la prog\u00e9rine. Ainsi, si le m\u00e9dicament FTI peut bloquer la fixation du groupe farn\u00e9syle sur la prog\u00e9rine chez les enfants atteints de prog\u00e9ria, la prog\u00e9rine pourrait alors \u00eatre \u00ab paralys\u00e9e \u00bb et la prog\u00e9ria am\u00e9lior\u00e9e.\u00a0\u00a0Cliquez ici<\/a>\u00a0pour plus d'informations sur les FTI.<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dComment le PRF a-t-il financ\u00e9 l\u2019essai ?\u201d _builder_version=\u201d4.16\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Gr\u00e2ce au soutien de milliers de personnes, nous avons pu r\u00e9unir tous les fonds n\u00e9cessaires pour couvrir les frais du proc\u00e8s. Nous exprimons notre plus sinc\u00e8re gratitude \u00e0 tous ceux qui ont consacr\u00e9 leur temps, leurs talents et leur argent \u00e0 rendre possible cette incroyable r\u00e9ussite, et bien s\u00fbr \u00e0 toutes les familles courageuses qui y ont particip\u00e9.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|35px|35.2344px|35px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dLe lonafarnib FTI est d\u00e9sormais un traitement \u00e9prouv\u00e9 contre la prog\u00e9ria.\u201d _builder_version=\u201d4.22.1\u2033 text_font=\u201d||||||||\u201d header_font=\u201d||||||||\u201d header_text_align=\u201dcentre\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201dsombre\u201d animation_style=\u201dfondu\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201dsur|bureau\u201d global_colors_info=\u201d{}\u201d]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201d2012 2014\u2033 _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d39px||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n En 2012, les r\u00e9sultats de l\u2019\u00e9tude ont \u00e9t\u00e9 publi\u00e9s, d\u00e9montrant que chaque enfant a connu une am\u00e9lioration dans un ou plusieurs domaines, y compris le syst\u00e8me cardiovasculaire vital. En mai 2014, une \u00e9tude a montr\u00e9 qu\u2019un ou plusieurs des 3 m\u00e9dicaments \u2013 dont le lonafarnib \u2013 test\u00e9s dans le cadre d\u2019essais cliniques financ\u00e9s par le PRF prolongeaient la dur\u00e9e de vie ; on ne savait pas quel m\u00e9dicament avait cet impact positif sur la vie. Cependant, en avril 2018, une \u00e9tude publi\u00e9e dans Journal de l'Association m\u00e9dicale am\u00e9ricaine (JAMA)<\/em> il a \u00e9t\u00e9 rapport\u00e9 que le lonafarnib seul prolongeait la survie des enfants atteints de prog\u00e9ria d'au moins 1,6 an. Cliquez ici<\/a>\u00a0<\/a>pour plus de d\u00e9tails sur l'\u00e9tude historique de d\u00e9couverte de traitement de 2012,\u00a0ici\u00a0<\/a>pour plus de d\u00e9tails sur les r\u00e9sultats de 2014, et\u00a0ici<\/a>\u00a0pour plus de d\u00e9tails sur l\u2019\u00e9tude de 2018.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 rembourrage_personnalis\u00e9=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_testimonial portrait_url=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cam-and-DadMarch09.jpg\u201d quote_icon_color=\u201d#00b2e2\u2033 portrait_width=\u201d140px\u201d portrait_height=\u201d140px\u201d _builder_version=\u201d4.16\u2033 background_color=\u201d#f7f7f7\u2033 custom_margin_tablet=\u201d\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 custom_css_testimonial_author=\u201dcolor: #00b2e2;||font-size: 19px !important;||font-weight: 700;\u201d custom_css_testimonial_meta=\u201dfont-style: italic;||font-weight: 700;\u201d box_shadow_style_image=\u201dpreset3\u2033 locked=\u201doff\u201d global_colors_info=\u201d{}\u201d]<\/p>\n \u00ab TOUT LE MONDE a \u00e9t\u00e9 si merveilleux. Pour nous, vous \u00eates TOUS ENVOY\u00c9S PAR DIEU et nous APPR\u00c9CIONS tout ce que vous faites pour ces petits anges. Notre famille est tellement submerg\u00e9e par l'excitation et toutes sortes d'\u00e9motions \u00e0 l'id\u00e9e du voyage d'Adalia \u00e0 Boston ce week-end, que je ne peux m\u00eame pas commencer \u00e0 \u00e9crire les mots pour exprimer ce que nous ressentons. \u00bb<\/em><\/p>\n \u00ab Ce nouveau m\u00e9dicament pour Zach nous redonne l\u2019espoir que son c\u0153ur sera plus fort, son sourire plus \u00e9clatant et sa vie plus longue. Ce nouveau m\u00e9dicament est une r\u00e9ponse \u00e0 nos pri\u00e8res. Merci \u00e0 tous ceux qui ont particip\u00e9 \u00e0 la PRF et qui ont rendu cela possible\u2026 les m\u00e9decins, les chercheurs et le personnel. Vous \u00eates nos h\u00e9ros ! \u00bb<\/em><\/p>\n \u00ab Au nom de Cam et de notre famille, merci beaucoup \u00e0 tous les membres de PRF pour tout ce que vous avez fait ! Sans vous, nous serions perdus dans un monde de confusion et de chagrin. Au lieu de cela, nous vivons dans un monde d\u2019espoir et de d\u00e9termination. Merci encore et encore ! Avec beaucoup d\u2019amour et de respect. \u00bb\u00a0<\/em><\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_margin=\u201d39px||\u201d custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201ddroite\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/trophies-3-kids.jpg\u201d align=\u201dcenter\u201d align_tablet=\u201dcenter\u201d align_phone=\u201d\u201d align_last_edited=\u201don|desktop\u201d admin_label=\u201dimage d\u2019enfants Aloha\u201d _builder_version=\u201d4.22.1\u2033 animation_style=\u201dfade\u201d animation_direction=\u201dbottom\u201d global_colors_info=\u201d{}\u201d] <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|30px|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dR\u00e9sum\u00e9, etc.\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d39px||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Les chercheurs ont identifi\u00e9 deux m\u00e9dicaments suppl\u00e9mentaires qui, utilis\u00e9s en association avec le m\u00e9dicament FTI actuellement test\u00e9 (lonafarnib), pourraient constituer un traitement encore plus efficace pour les enfants atteints de prog\u00e9ria que le FTI seul. La pravastatine et le zol\u00e9dronate ont \u00e9t\u00e9 ajout\u00e9s au traitement actuel, le lonafarnib. Cet essai beaucoup plus vaste a port\u00e9 sur 45 enfants de 24 pays diff\u00e9rents !<\/p>\n Ces trois m\u00e9dicaments ciblent des points diff\u00e9rents le long de la voie conduisant \u00e0 la production de la prog\u00e9rine, responsable de la maladie. Dans le cadre d'\u00e9tudes de laboratoire passionnantes pr\u00e9sent\u00e9es par le Dr Carlos Lopez-Otin d'Espagne lors de l'atelier scientifique 2007 de la Progeria Research Foundation, les deux nouveaux m\u00e9dicaments ont am\u00e9lior\u00e9 la maladie dans les cellules prog\u00e9ria et prolong\u00e9 la dur\u00e9e de vie dans les mod\u00e8les murins de prog\u00e9ria.<\/p>\n Si les trois m\u00e9dicaments administr\u00e9s dans cet essai peuvent bloquer efficacement cette fixation du groupe farn\u00e9syl, alors la prog\u00e9rine pourrait \u00eatre \u00ab paralys\u00e9e \u00bb et la prog\u00e9ria pourrait \u00eatre am\u00e9lior\u00e9e encore plus qu\u2019avec le lonafarnib seul. On esp\u00e8re que les m\u00e9dicaments fonctionneront comme des partenaires, pour se compl\u00e9ter mutuellement, de sorte que la prot\u00e9ine prog\u00e9rine soit davantage affect\u00e9e par la combinaison des trois m\u00e9dicaments.<\/p>\n L\u2019\u00e9quipe a men\u00e9 un mini-essai sur cinq enfants atteints de prog\u00e9ria. L\u2019essai de \u00ab faisabilit\u00e9 \u00bb d\u2019un mois visait \u00e0 d\u00e9terminer si la combinaison de trois m\u00e9dicaments serait bien tol\u00e9r\u00e9e, avant de se lancer dans un essai international de plus grande envergure. Les effets secondaires se sont r\u00e9v\u00e9l\u00e9s acceptables et l\u2019\u00e9quipe a proc\u00e9d\u00e9 \u00e0 un essai d\u2019efficacit\u00e9 de plus grande envergure.\u00a0\u00a0<\/strong><\/p>\n 45 enfants ont particip\u00e9 \u00e0 cet essai, originaires de 24 pays et parlant 17 langues. Cela comprend les enfants qui ont particip\u00e9 \u00e0 l'essai FTI uniquement, les participants \u00e0 l'essai de faisabilit\u00e9 et d'autres enfants qui \u00e9taient soit trop jeunes pour participer au premier essai, soit des enfants que nous avons d\u00e9couverts au cours du premier essai clinique (apr\u00e8s la fin du recrutement). Les enfants inscrits \u00e0 l'essai FTI uniquement ont eu la possibilit\u00e9 de s'inscrire \u00e0 l'essai triple lors de leur derni\u00e8re visite pour l'essai en cours. Cela a permis \u00e0 ces enfants de continuer \u00e0 prendre FTI sans manquer aucune dose.<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dLes m\u00e9dicaments d\u2019essai en un coup d\u2019\u0153il\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d39px||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Pravastatine<\/strong>\u00a0(commercialis\u00e9 sous le nom de Pravachol ou Selektine) fait partie de la classe des m\u00e9dicaments appel\u00e9s statines. Il est g\u00e9n\u00e9ralement utilis\u00e9 pour r\u00e9duire le cholest\u00e9rol et pr\u00e9venir les maladies cardiovasculaires.<\/p>\n Acide zol\u00e9dronique\u00a0<\/strong>est un\u00a0bisphosphonate<\/strong>, g\u00e9n\u00e9ralement utilis\u00e9 comme m\u00e9dicament osseux pour am\u00e9liorer l\u2019ost\u00e9oporose et pour pr\u00e9venir les fractures squelettiques chez les personnes souffrant de certaines formes de cancer.<\/p>\n Lonafarnib <\/strong>est un\u00a0FTI\u00a0<\/strong>(Inhibiteur de la farn\u00e9syltransf\u00e9rase), un m\u00e9dicament qui peut inverser une anomalie dans les cellules Progeria en laboratoire, et qui a am\u00e9lior\u00e9 la maladie chez les souris Progeria. En juillet un\u00a0\u00e9tude\u00a0<\/a>** a \u00e9t\u00e9 publi\u00e9 et n'a montr\u00e9 aucune am\u00e9lioration significative par rapport \u00e0 la monoth\u00e9rapie par lonafarnib. **Gordon, et. al., Essai clinique des inhibiteurs de la farn\u00e9sylation des prot\u00e9ines lonafarnib, pravastatine et acide zol\u00e9dronique chez les enfants atteints du syndrome de Hutchinson-Gilford-Progeria, Circulation<\/em>, 10.1161\/CIRCULATIONAHA.116.022188<\/strong><\/p>\n Cependant, le \u00ab Triple essai \u00bb a \u00e9t\u00e9 prolong\u00e9 au-del\u00e0 de sa dur\u00e9e initiale de 2 \u00e0 3 ans et a \u00e9t\u00e9 \u00e9largi pour inclure jusqu\u2019\u00e0 80 enfants, afin que chaque enfant puisse avoir acc\u00e8s au lonafarnib seul, car nous savons que ce m\u00e9dicament les aide. Habituellement, les essais cliniques suivent leur cours et les patients sont priv\u00e9s de tous les m\u00e9dicaments jusqu\u2019\u00e0 l\u2019approbation de la FDA, ce qui peut prendre des ann\u00e9es. La PRF a veill\u00e9 \u00e0 ce que les enfants continuent de prendre le seul traitement connu, tandis qu\u2019elle et ses partenaires de recherche continuent d\u2019explorer d\u2019autres options de traitement (comme l\u2019\u00e9v\u00e9rolimus qui est actuellement test\u00e9).<\/p>\n [\/et_pb_text][et_pb_text admin_label=\u201dEverolimus\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d||25px\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201don|phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n L\u2019\u00e9v\u00e9rolimus est une forme de rapamycine, un m\u00e9dicament qui pourrait \u00eatre administr\u00e9 plus facilement aux enfants atteints de prog\u00e9ria, car il n\u00e9cessite moins de prises de sang pour mesurer les concentrations du m\u00e9dicament. Alors que le lonafarnib peut bloquer le d\u00e9veloppement de la prog\u00e9rine toxique, la rapamycine semble permettre aux cellules d\u2019\u00e9liminer plus rapidement la prog\u00e9rine. Ainsi, la rapamycine ciblant une voie diff\u00e9rente de celle du lonafarnib, la combinaison pourrait s\u2019av\u00e9rer \u00eatre un \u00ab coup double \u00bb contre la prog\u00e9ria \u2013 et, esp\u00e9rons-le, un meilleur traitement que le lonafarnib seul.<\/p>\n Rapamycin<\/em> est un m\u00e9dicament approuv\u00e9 par la FDA qui a d\u00e9j\u00e0 montr\u00e9 qu'il prolongeait la vie des mod\u00e8les de souris non atteintes de la prog\u00e9ria. Une \u00e9tude* men\u00e9e par des chercheurs du NIH \u00e0 Bethesda, dans le Maryland, et du Massachusetts General Hospital \u00e0 Boston d\u00e9montre que la rapamycine diminue la quantit\u00e9 de la prot\u00e9ine prog\u00e9rine responsable de la maladie de 50%, am\u00e9liore la forme nucl\u00e9aire anormale et prolonge la dur\u00e9e de vie des cellules de la prog\u00e9ria en laboratoire.<\/p>\n La rapamycine est connue pour ses propri\u00e9t\u00e9s anti-\u00e2ge chez la souris. Ces r\u00e9sultats font partie d\u2019une liste croissante d\u2019\u00e9tudes qui contribuent \u00e0 valider la th\u00e9orie selon laquelle la d\u00e9couverte d\u2019un rem\u00e8de contre la prog\u00e9ria pourrait \u00e9galement b\u00e9n\u00e9ficier \u00e0 l\u2019ensemble de la population vieillissante.<\/p>\n \u00a0* K. Cao, J. J. Graziotto, C. D. Blair, J. R. Mazzulli, M. R. Erdos, D. Krainc, F. S. Collins, \u00ab La rapamycine inverse les ph\u00e9notypes cellulaires et am\u00e9liore la clairance des prot\u00e9ines mutantes dans les cellules atteintes du syndrome de Hutchinson-Gilford prog\u00e9ria. \u00bb Sci. Transl. Med. 3, 89ra58 (2011).<\/p>\n La Fondation de recherche sur la prog\u00e9ria a fourni des cellules pour ce projet \u00e0 partir du\u00a0Banque de cellules et de tissus PRF<\/a>\u00a0et a aid\u00e9 \u00e0 financer la recherche gr\u00e2ce \u00e0 notre\u00a0programme de subventions<\/a>\u00a0\u2013 une preuve suppl\u00e9mentaire que les programmes de recherche du PRF sont essentiels aux progr\u00e8s vers la gu\u00e9rison.<\/p>\n Cet essai portant sur deux m\u00e9dicaments est le fruit d'une collaboration qui s'appuie sur les connaissances acquises lors des pr\u00e9c\u00e9dents essais cliniques du PRF. Les enfants ont \u00e9t\u00e9 suivis par pratiquement la m\u00eame \u00e9quipe de m\u00e9decins du Boston Children's Hospital et du Brigham and Women's Hospital, qui poss\u00e8dent tous d\u00e9sormais une expertise reconnue mondialement dans le domaine de la prog\u00e9ria ainsi que dans les m\u00e9dicaments impliqu\u00e9s.<\/p>\n Soixante enfants de 27 pays ont \u00e9t\u00e9 recrut\u00e9s dans cette phase \u00e0 deux m\u00e9dicaments. Les donn\u00e9es de la partie de l'essai \u00e0 deux m\u00e9dicaments sont en cours d'analyse et les r\u00e9sultats sont en cours de formulation et de r\u00e9daction en vue d'une publication dans une revue scientifique \u00e0 comit\u00e9 de lecture.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|30px|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px|2px|35px|false|false\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201ddroite\u201d border_width_top=\u201d10px\u201d border_color_top=\u201d#8fd2ed\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#00b2e2\u2033 global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dQu\u00eate\u201d _builder_version=\u201d4.22.1\u2033 header_4_line_height=\u201d1.2em\u201d custom_margin=\u201d25px||25px\u201d custom_padding=\u201d||\u201d animation_direction=\u201ddroite\u201d header_4_font_size_tablet=\u201d\u201d header_4_font_size_phone=\u201d23px\u201d header_4_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n Nos travaux sur les th\u00e9rapies g\u00e9n\u00e9tiques avancent \u00e0 grands pas ! \u00c9tudes sur la th\u00e9rapie par ARN et l'\u00e9dition g\u00e9n\u00e9tique de l'ADN <\/strong>ont montr\u00e9 une vaste am\u00e9lioration de la dur\u00e9e de vie des souris atteintes de Progeria. <\/strong>PRF continue d\u2019investir des fonds substantiels dans leur d\u00e9veloppement, avec l\u2019espoir que ces efforts de recherche conduire \u00e0 des essais cliniques et, finalement, au rem\u00e8de.<\/strong><\/p>\n Ces th\u00e9rapies de pointe ont \u00e9norme potentiel<\/em>! Avec votre aide, PRF peut continuer \u00e0 progresser le plus rapidement possible vers les traitements les plus efficaces et la gu\u00e9rison.<\/p>\n [\/et_pb_text][et_pb_button button_url=\u201dhttps:\/\/www.progeriaresearch.org\/lonafarnib-map-program\/\u201d button_text=\u201dPatients, soignants et m\u00e9decins : cliquez ici pour plus de d\u00e9tails.\u201d admin_label=\u201dBouton du PDF de Lonafarnib\u201d _builder_version=\u201d4.22.1\u2033 background_layout=\u201ddark\u201d custom_margin=\u201d10px||10px||false|false\u201d z_index_tablet=\u201d500\u2033 button_text_shadow_horizontal_length_tablet=\u201d0px\u201d button_text_shadow_vertical_length_tablet=\u201d0px\u201d button_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d box_shadow_spread_tablet=\u201d0px\u201d global_colors_info=\u201d{}\u201d] [\/et_pb_cta][\/et_pb_column][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_cta button_url=\u201dhttps:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR\u201d button_text=\u201dFaites un don maintenant\u201d admin_label=\u201dEnsemble, nous trouverons le rem\u00e8de !\u201d module_class=\u201dsign-btn\u201d _builder_version=\u201d4.16\u2033 header_font_size=\u201d25px\u201d background_color=\u201d#29327a\u201d animation_style=\u201dslide\u201d animation_direction=\u201dleft\u201d animation_intensity_slide=\u201d25%\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d body_font_size_tablet=\u201d\u201d body_font_size_phone=\u201d\u201d body_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 border_radii=\u201don|25px|25px|25px|25px\u201d global_colors_info=\u201d{}\u201d button_bg_color__hover_enabled=\u201don\u201d bouton_bg_color__hover=\u201d#8fd2ed\u201d bouton_border_color__hover_enabled=\u201don\u201d]<\/p>\n [\/et_pb_cta][\/et_pb_column][et_pb_column type=\u201d1_2\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2024\/08\/2024-strip-footer-strip-copy.png\u201d title_text=\u201dCopie de la bande de pied de page de la bande 2024\u201d _builder_version=\u201d4.27.0\u2033 _module_preset=\u201ddefault\u201d custom_margin=\u201d35px||||false|false\u201d global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column][\/et_pb_row][\/et_pb_section]<\/p>","protected":false},"excerpt":{"rendered":" [et_pb_section fb_built=\u201d1\u2033 fullwidth=\u201don\u201d disabled_on=\u201doff|off|off\u201d _builder_version=\u201d4.16\u2033 border_width_bottom=\u201d55px\u201d border_color_bottom=\u201d#29327a\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55px\u201d background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/09\/Kids-on-Bridge.png\u201d custom_padding=\u201d11.5vw||11.5vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited=\u201dsur|bureau\u201d title_font_size_tablet=\u201d45px\u201d title_font_size_phone=\u201d40px\u201d title_font_size_last_edited=\u201dsur|t\u00e9l\u00e9phone\u201d z_index_tablet=\u201d500\u2033 custom_css_main_element=\u201dbackground-position: center 18% !important;\u201d title_text_shadow_horizontal_length_tablet=\u201d0px\u201d title_text_shadow_vertical_length_tablet=\u201d0px\u201d title_text_shadow_blur_strength_tablet=\u201d1px\u201d content_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_text_shadow_vertical_length_tablet=\u201d0px\u201d content_text_shadow_blur_strength_tablet=\u201d1px\u201d subhead_text_shadow_horizontal_length_tablet=\u201d0px\u201d subhead_text_shadow_vertical_length_tablet=\u201d0px\u201d subhead_text_shadow_blur_strength_tablet=\u201d1px\u201d content_link_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_link_text_shadow_vertical_length_tablet=\u201d0px\u201d content_link_text_shadow_blur_strength_tablet=\u201d1px\u201d content_ul_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_ul_text_shadow_vertical_length_tablet=\u201d0px\u201d content_ul_text_shadow_blur_strength_tablet=\u201d1px\u201d content_ol_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_ol_text_shadow_vertical_length_tablet=\u201d0px\u201d content_ol_text_shadow_blur_strength_tablet=\u201d1px\u201d content_quote_text_shadow_horizontal_length_tablet=\u201d0px\u201d content_quote_text_shadow_vertical_length_tablet=\u201d0px\u201d content_quote_text_shadow_blur_strength_tablet=\u201d1px\u201d button_one_text_shadow_horizontal_length_tablet=\u201d0px\u201d button_one_text_shadow_vertical_length_tablet=\u201d0px\u201d button_one_text_shadow_blur_strength_tablet=\u201d1px\u201d button_two_text_shadow_horizontal_length_tablet=\u201d0px\u201d button_two_text_shadow_vertical_length_tablet=\u201d0px\u201d button_two_text_shadow_blur_strength_tablet=\u201d1px\u201d box_shadow_horizontal_tablet=\u201d0px\u201d box_shadow_vertical_tablet=\u201d0px\u201d box_shadow_blur_tablet=\u201d40px\u201d [\u2026]<\/p>","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_et_pb_use_builder":"on","_et_pb_old_content":"\t\t\t\t[vc_row][vc_column][vc_custom_heading text=\"Clinical Trials\" font_container=\"tag:h1|text_align:center\" use_theme_fonts=\"yes\"][vc_column_text]From total obscurity in 1999 to first-ever clinical drug trials starting in 2007, PRF is leading the charge in the quest to discover treatments and the cure.\r\n The FTI lonafarnib is now a proven treatment for Progeria. In 2012 the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.\u00a0 And in May 2014, further study revealed lonafarnib (and possibly the other 2 drugs tested in the Triple Trial \u2013 see below) increases estimated lifespan by at least 1.6 years (time will tell if that number increases \u2013 it just hasn\u2019t been long enough to determine.)\u00a0 Click here<\/a> for details on the 2012 study, and here<\/a> for details on the 2014 findings.<\/p>\r\n<\/a>\r\nAlways Moving Forward: The Progeria Triple Drug Trial Begins August 2009<\/strong>\r\n\r\nWho Enrolled in the Triple Drug Trial?<\/b>\r\n\r\nThe Progeria Research Foundation and Boston Children\u2019s Hospital once again partnered to conduct a new clinical trial for children with Progeria. This much larger trial initially included 45 children from 24 different countries!\r\n\r\n\u00a0\r\n\r\n[caption id=\"\" align=\"alignright\" width=\"200\"] \u201cOn behalf of Cam and our family, thank you all at PRF so much for all you have done! We would have been lost in a world of confusion and grief without you. Instead, we live in a world of hope and purpose. Thank you again and again! With much love and respect,\u201d\u00a0<\/em><\/p>\r\nThere were changes to the Triple Trial protocol.<\/b>\r\n\r\n[caption id=\"\" align=\"alignright\" width=\"150\"]Lancement d'un nouvel essai clinique de m\u00e9dicament : le recrutement pour l'essai sur la prog\u00e9rinine a commenc\u00e9 !<\/em><\/strong><\/h4>\n
E-mail: sphillips@progeriaresearch.org<\/a>
WhatsApp, Telegram, WeChat : 1-978-876-2407
T\u00e9l\u00e9phone du bureau : 978-548-5308<\/p>\nTh\u00e9rapie par ARN : administration des m\u00e9dicaments<\/strong> L\u2019\u00e9tude de faisabilit\u00e9 est termin\u00e9e !<\/strong><\/h4>\n
Que se passe-t-il AUJOURD'HUI avec les essais cliniques sur la prog\u00e9ria ?<\/strong><\/h2>\n
Aper\u00e7u de l'historique du proc\u00e8s<\/b><\/h2>\n
\n[\/et_pb_button][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201d1_2,1_2\u2033 custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_padding=\u201d0|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d0px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201d1_2\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dD\u00e9tails sur les essais pr\u00e9c\u00e9dents\u201d _builder_version=\u201d4.27.0\u2033 global_colors_info=\u201d{}\u201d]<\/p>\nD\u00e9tails sur les proc\u00e8s ant\u00e9rieurs<\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d custom_padding_last_edited=\u201don|desktop\u201d padding_left_right_link_1=\u201dtrue\u201d padding_left_right_link_2=\u201dtrue\u201d _builder_version=\u201d4.16\u2033 custom_margin=\u201d||25px\u201d custom_padding=\u201d|35px|0|0px|false|false\u201d custom_padding_tablet=\u201d35px|35px||35px\u201d custom_padding_phone=\u201d0px||0px\u201d animation_direction=\u201dright\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201d7 mai 2007 : Le d\u00e9but du tout premier essai clinique sur le m\u00e9dicament contre la prog\u00e9ria marque un moment historique dans l\u2019histoire de la recherche sur la prog\u00e9ria !\u201d _builder_version=\u201d4.27.2\u2033 z_index_tablet=\u201d500\u2033 global_colors_info=\u201d{}\u201d]<\/p>\n7 mai 2007 : Le d\u00e9but du tout premier essai clinique du m\u00e9dicament contre la prog\u00e9ria marque un moment historique dans l'histoire de la recherche sur la prog\u00e9ria !<\/strong><\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=\u201d1_2\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|desktop\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dVingt-huit (28) enfants de seize pays ont particip\u00e9\u201d _builder_version=\u201d4.27.0\u2033 custom_margin=\u201d|||-30px|false|false\u201d custom_margin_tablet=\u201d35px||\u201d custom_margin_phone=\u201d\u201d custom_margin_last_edited=\u201dsur|bureau\u201d global_colors_info=\u201d{}\u201d]<\/p>\nQui, O\u00f9, Quand, Comment et Combien\u2026<\/strong><\/h2>\n
Comment en sommes-nous arriv\u00e9s l\u00e0 ?<\/b><\/h4>\n
Pourquoi les chercheurs pensaient-ils que ce m\u00e9dicament fonctionnerait contre la prog\u00e9ria ?<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/FTI-3-cell-image-crpd-300x71.jpg\")
Comment le PRF a-t-il financ\u00e9 le proc\u00e8s ?
<\/strong><\/h4>\nLe lonafarnib de FTI est d\u00e9sormais un traitement \u00e9prouv\u00e9 contre la prog\u00e9ria<\/strong>.<\/h2>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=\u201dundefined\u201d use_custom_gutter=\u201don\u201d gutter_width=\u201d1\u2033 module_id=\u201dthreeCT\u201d _builder_version=\u201d4.16\u2033 background_color=\u201d#29327a\u201d custom_padding=\u201d40px|0px|35.2344px|0px|false|false\u201d animation_style=\u201dslide\u201d animation_direction=\u201dright\u201d animation_intensity_slide=\u201d25%\u201d border_width_bottom=\u201d10px\u201d border_color_bottom=\u201d#8fd2ed\u201d locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201dundefined\u201d saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d custom_padding_tablet=\u201d|0px||0px\u201d custom_padding_phone=\u201d||30px\u201d custom_padding_last_edited=\u201don|phone\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_text admin_label=\u201dL\u2019essai clinique du triple m\u00e9dicament contre la prog\u00e9ria commence en ao\u00fbt 2009\u2033 _builder_version=\u201d4.22.1\u2033 text_font=\u201d||||||||\u201d header_font=\u201d||||||||\u201d header_text_align=\u201dcentre\u201d header_text_color=\u201d#ffffff\u201d background_layout=\u201dsombre\u201d animation_style=\u201dfondu\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201dsur|bureau\u201d global_colors_info=\u201d{}\u201d]<\/p>\nToujours en marche vers l'avant : l'essai clinique du triple m\u00e9dicament contre la prog\u00e9ria d\u00e9bute en ao\u00fbt 2009<\/strong><\/h2>\n
R\u00e9sum\u00e9:<\/strong><\/h4>\n
Strat\u00e9gie:<\/strong><\/h4>\n
But:<\/strong><\/h4>\n
L'essai de faisabilit\u00e9 :<\/strong><\/h4>\n
L'essai d'efficacit\u00e9 :<\/strong><\/h4>\n
Aper\u00e7u des m\u00e9dicaments \u00e0 l'essai<\/strong><\/h4>\n
Les trois m\u00e9dicaments bloquent la production de la mol\u00e9cule de farn\u00e9syle n\u00e9cessaire \u00e0 la prog\u00e9rine pour cr\u00e9er la maladie de Progeria.
<\/em>
*<\/em>\u00a0\u00ab<\/strong>Un traitement combin\u00e9 \u00e0 base de statines et d'aminobisphosphonates prolonge la long\u00e9vit\u00e9 dans un mod\u00e8le murin de vieillissement pr\u00e9matur\u00e9 humain"<\/strong>, par Ignacio Varela, Sandrine Pereira, Alejandro P. Ugalde, Claire L. Navarro, Mar\u00eda F. Suarez, Pierre Cau, Juan Cadinanos, Fernando G. Osorio, Nicolas Foray, Juan Cobo, Felix de Carlos, Nicolas Levy, Jose Le d\u00e9put\u00e9 Freije et Carlos Lopez-Otan. M\u00e9decine naturelle, 2008. 14(7) : p. 767-72.<\/p>\nL'ajout d'un plus r\u00e9cent<\/strong> M\u00e9dicament : Everolimus<\/strong><\/h4>\n
La science derri\u00e8re l\u2019ajout de ce deuxi\u00e8me m\u00e9dicament<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cao-with-caption.jpg\")
<\/em><\/p>\nNotre qu\u00eate du rem\u00e8de continue\u2026<\/strong><\/h4>\n
\n[\/et_pb_button][\/et_pb_column_inner][\/et_pb_row_inner][\/et_pb_column][\/et_pb_section][et_pb_section fb_built=\u201d1\u2033 module_class=\u201dfooter\u201d _builder_version=\u201d4.21.0\u2033 background_color=\u201d#29327a\u201d custom_margin=\u201d-2px|||||\u201d custom_padding=\u201d0|0px|0|0px|false|false\u201d z_index_tablet=\u201d500\u2033 border_width_top=\u201d12px\u201d border_color_top=\u201d#00b2e2\u2033 global_module=\u201d133\u2033 locked=\u201doff\u201d global_colors_info=\u201d{}\u201d][et_pb_row_inner column_structure=\u201d1_3,1_3,1_3\u2033 disabled_on=\u201don|on|on\u201d admin_label=\u201dRow\u201d _builder_version=\u201d4.21.0\u2033 _module_preset=\u201ddefault\u201d min_height=\u201d277px\u201d custom_margin=\u201d|20px|30px||false|false\u201d custom_padding=\u201d0px|30px|0px||false|false\u201d disabled=\u201don\u201d global_colors_info=\u201d{}\u201d][et_pb_column_inner type=\u201d1_3\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.21.0\u2033 _module_preset=\u201dpar d\u00e9faut\u201d global_colors_info=\u201d{}\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/08\/Startbanner-1.png\u201d title_text=\u201dStartbanner\u201d url=\u201dhttps:\/\/www.gravoc.com\/\u201d align=\u201dright\u201d _builder_version=\u201d4.21.0\u2033 _module_preset=\u201dpar d\u00e9faut\u201d custom_margin=\u201d|-50px||20px|false|false\u201d global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=\u201d1_3\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.21.0\u2033 _module_preset=\u201ddefault\u201d global_colors_info=\u201d{}\u201d][\/et_pb_column_inner][et_pb_column_inner type=\u201d1_3\u2033 saved_specialty_column_type=\u201d3_4\u2033 _builder_version=\u201d4.21.0\u2033 _module_preset=\u201ddefault\u201d global_colors_info=\u201d{}\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/07\/RFRFinish.png\u201d title_text=\u201dRFRFinish\u201d _builder_version=\u201d4.21.0\u2033 _module_preset=\u201ddefault\u201d custom_margin=\u201d|20px||-50px|false|false\u201d global_colors_info=\u201d{}\u201d][\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row column_structure=\u201d1_4,1_4,1_2\u2033 make_equal=\u201don\u201d module_class=\u201d et_pb_row_fullwidth\u201d _builder_version=\u201d4.16\u2033 width=\u201d89%\u201d width_tablet=\u201d80%\u201d width_phone=\u201d\u201d width_last_edited=\u201don|desktop\u201d max_width=\u201d89%\u201d max_width_tablet=\u201d80%\u201d max_width_phone=\u201d\u201d max_width_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 make_fullwidth=\u201don\u201d width_unit=\u201doff\u201d custom_width_percent=\u201d100%\u201d global_colors_info=\u201d{}\u201d][et_pb_column type=\u201d1_4\u2033 _builder_version=\u201d4.16\u2033 custom_padding=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_cta button_url=\u201dhttps:\/\/www.progeriaresearch.org\/newsletter-signup\/\u201d button_text=\u201dInscrivez-vous maintenant\u201d admin_label=\u201dInscrivez-vous \u00e0 la newsletter\u201d module_class=\u201dsign-btn\u201d _builder_version=\u201d4.16\u2033 header_font_size=\u201d25px\u201d background_color=\u201d#29327a\u201d animation_style=\u201dslide\u201d animation_direction=\u201dleft\u201d animation_intensity_slide=\u201d25%\u201d header_font_size_tablet=\u201d\u201d header_font_size_phone=\u201d30px\u201d header_font_size_last_edited=\u201don|desktop\u201d z_index_tablet=\u201d500\u2033 border_radii=\u201dactiv\u00e9|25px|25px|25px|25px\u201d global_colors_info=\u201d{}\u201d button_bg_color__hover_enabled=\u201dactiv\u00e9\u201d button_bg_color__hover=\u201d#8fd2ed\u201d button_border_color__hover_enabled=\u201dactiv\u00e9\u201d]<\/p>\nS'inscrire<\/h2>\n
pour notre<\/h2>\n
Bulletin!<\/h2>\n
Ensemble, nous<\/h2>\n
VOLONT\u00c9<\/em><\/h2>\n
trouve le rem\u00e8de !<\/h2>\n
Trials history at-a-glance:<\/strong><\/h4>\r\nIn just 17 years, since we founded PRF and there were no resources for these children, we have soared from total obscurity, to gene finding, to the first clinical trials in Progeria, to a first-ever treatment \u2013 all at a pace virtually unheard of in the scientific community. And while helping this handful of children, the connection of Progeria to common heart disease and aging has tremendous implications for us all.\r\n\r\nTo date, PRF has funded and co-coordinated four clinical trials. PRF is and always has been responsible for\u00a0all trial expenses, including testing, travel, food, lodging, translators and staff.\u00a0\u00a0 Each new trial is more expensive than the last, as more children enroll for a chance at longer, healthier lives. Please help us fund these vital trials \u2013 DONATE TODAY!<\/a>\r\n\r\n#1<\/strong> involved a single drug lonafarnib, began in 2007, and proved successful;\u00a0<\/a>\r\n\r\n#2<\/strong> was a 1-month, phase 1 \u201cmini trial\u201d in March 2009 to determine if adding 2 more drugs to the lonafarnib regimen (pravastatin and zoledronate) was safe to move forward with a larger population (which it was);<\/a>\r\n\r\n#3<\/strong>, the \u201cTriple Trial\u201d involved the 3 drugs described above (lonafarnib, pravastatin and zoledronate) and began August 2009.\u00a0 Its protocol changed over the course of 5 years, switching back to just lonafarnib and re-opening enrollment so more children could participate; and<\/a>\r\n\r\n#4<\/strong> is a 2-drug trial involving the treatment lonafarnib and a new drug everolimus.\u00a0 It began in April 2016 and is ongoing.\u00a0 Phase 1, to determine the dosage of everolimus, was successfully completed in June 2017. Phase 2, which will test the effectiveness of the 2-drug combination, began in July 2017.\u00a0 As of February 2018, 41 children from 18 countries have enrolled.\u00a0<\/a>\r\n\r\nClick here to learn more about the current clinical trial.<\/a>\r\n\r\nRead all about the historic treatment discovery here,\u00a0<\/a>and below is the detailed history of these 4 trials.[\/vc_column_text][vc_column_text]<\/a>May 7, 2007:\u00a0The Start of the First-Ever Progeria Clinical Drug Trial Marks Historic Moment in Progeria Research History!<\/strong>\r\n
![](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/2-megans-frnt-cover.jpg\")
\r\n\r\nIn 2006, researchers identified\u00a0a potential\u00a0drug treatment\u00a0for children with\u00a0Progeria,\u00a0called FTIs.<\/a>\u00a0For the first time, we had in front of us a possible treatment for children with Progeria.\u00a0<\/b>Exciting times! \u00a0The Progeria clinical drug trial began on May 7th, 2007 with two children \u2013 Meghan and Megan - arriving at Boston Children\u2019s Hospital in Boston, MA for their first of seven visits over a 2-year period. At this first visit, they were given extensive tests and their first doses of the drug. An average of two families traveled to Boston each week thereafter, through December 2009, followed by a period of time in which the trial team analyzed the many thousands of data elements (each child underwent over 100 tests per visit!)and sought publication of the results.\r\n\r\n\u201cI know of no other rare genetic disease that has gone from gene discovery to clinical trial in under four years - a phenomenal testament to the hard work of The Progeria Research Foundation.\u201d \u00a0Francis Collins, MD, PhD, Director of the National Human Genome Research Institute that mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.<\/i>\r\n\r\nTwenty-eight (28) children from sixteen countries participated, ages 3 to 15 years.\u00a0Children returned to Children\u2019s Hospital Boston every four months, for testing and to receive new drug supply, and stayed in Boston for 4-8 days each visit. \u00a0While at home, their doctors kept a close watch over the children and submitted periodic health reports to the Boston research team.\u00a0 For the duration of the trial, an average of 2 children per week traveled to Boston to participate.\r\n![\"Children](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/Trial-kids-words-flags.jpg\")
<\/p>\r\nThe Progeria Clinical Drug Trial:\u00a0 Who, Where, When, How and How Much\u2026<\/strong>\r\n\r\nThe first three clinical trials were led by Mark Kieran MD, PhD, <\/b>Director, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute and Children\u2019s Hospital Boston; Assistant Professor, Departments of Pediatrics and Hematology\/Oncology, Harvard Medical School.\u00a0Dr. Kieran is a pediatric oncologist with extensive experience with the drug under study (farnesyltransferase, or FTI) in children. Co-Chairs were Monica Kleinman, MD, Director of Medical-Surgical Intensive Care Unit, Sr. Associate in Critical Care Medicine at BCH, Assistant Prof. at Harvard Medical School; and\u00a0 Leslie Gordon, MD, PhD, Medical Director of PRF, Lecturer at BCH and Harvard Medical School, Associate Prof. of Pediatrics at Hasbro Children's Hospital and Brown University in Providence, RI.\u00a0 All three continue to lead the current trial, with Dr. Kleinman assuming the lead role of Principal Investigator.\u00a0The clinical trials are a collaborative effort, involving physicians at Boston Children\u2019s Hospital, Dana-Farber Cancer Institute, and Brigham and Women\u2019s Hospital,<\/b> all Harvard University institutions.\u00a0In addition, physicians and scientists from The Warren Alpert Medical School at Brown University and NIH helped to make this first and the other trials a success.\r\n\r\nHow did we get to this point?<\/b>\r\n\r\nIn 2003, The Progeria Research Foundation\u2019s collaborative research team\u00a0 discovered the Progeria gene<\/a>.<\/a>\u00a0\u00a0This discovery not only led to further understanding of Progeria, but scientists now know that studying Progeria can help us learn more about heart disease and the normal aging process that affects us all. \u00a0 Since the\u00a0gene discovery, the support of researchers, clinicians, families of children with Progeria and people like\u00a0YOU brought us to another crossroads in the search for a treatment. Researchers began an intense study of this enemy protein called progerin<\/i>, and in 2006 they identified\u00a0a potential\u00a0drug treatment\u00a0for children with\u00a0Progeria,\u00a0called farnesyltransferase inhibitors (FTIs), and conducted studies in the lab that supported a human trial with the drug. The FTI chosen is supplied by Merck and called lonafarnib<\/i>. \u00a0Click here<\/a>\u00a0for more details on the research.\r\n\r\nWhy did researchers think this drug would work in Progeria?<\/b>\r\n\r\nThe protein that we believe is responsible for Progeria is called progerin.\u00a0In order to block normal cell function and cause Progeria, a molecule called a \u201cfarnesyl group\u201d must be attached to the progerin protein. FTIs act by blocking (inhibiting) the attachment of the farnesyl group onto progerin. \u00a0So if the FTI drug can block this farnesyl group attachment in children with Progeria, then progerin may be \u201cparalyzed\u201d and Progeria improved.\u00a0Click here<\/a> for more information on FTIs.\r\n\r\n[caption id=\"\" align=\"aligncenter\" width=\"375\"]![\"Normal](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/FTI-3-cell-image-crpd.jpg\")
Normal cell, Progeria cell, Progeria cell after being treated with FTI.<\/strong><\/span>[\/caption]\r\n\r\nHow did PRF fund the trial? <\/b>\r\n\r\nThanks to the support of thousands, we were able to raise all the funds necessary to cover the trial costs, including clinical testing, translators, staff, travel, food and lodging for the 28 families from 16 countries who came to Boston every four months for two years.\u00a0 Our heartfelt gratitude goes out to everyone who contributed their \u201ctime, talents and treasure\u201d to make this incredible achievement possible, and of course to all the courageous families who participated.\r\n\r\n[caption id=\"\" align=\"aligncenter\" width=\"300\"]![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/trophies-3-kids.jpg\")
All of the children received trophies at their final, 2-year visit for the first-ever Progeria clinical drug trial. Here, Mateo, Milagros and Jesper are thrilled to get their awards<\/span>.<\/strong><\/span>[\/caption]\r\n\r\n\u00a0\r\n![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/HayleyMichielAmber-Aug09.jpg\")
Hayley from England and Michiel from Belgium are all smiles as they pose with their trophies for completing the first-ever Progeria clinical drug trial in August, 2009. They also, along with Michiel\u2019s sister Amber (right), completed their first visit for the triple drug trial that week.<\/strong><\/span>[\/caption]\r\n\r\n\u00a0\r\n\r\nSummary:<\/b>\u00a0Researchers identified two additional drugs that, when used in combination with the current FTI drug being tested (lonafarnib), may provide an even more effective treatment for children with Progeria than FTI\u2019s alone. Pravastatin and zoledronate were added to the current treatment lonafarnib.\r\n\r\nStrategy:<\/i><\/b> All three drugs target different points along the pathway leading to production of the disease-causing progerin. In exciting laboratory studies presented by Dr. Carlos Lopez-Otin of Spain at the 2007 Progeria Research Foundation Scientific Workshop, the two new drugs improved disease in Progeria cells and extended lifespan in mouse models of Progeria.\r\n\r\nGoal:<\/i><\/b> If the three drugs administered in this trial can effectively block this farnesyl group attachment, then progerin may be \u201cparalyzed\u201d and Progeria may be improved even more than it is with the lonafarnib alone. The hope is that the drugs will work as partners, to complement each other so that the progerin protein is affected more by combining the three drugs.\r\n\r\nThe Feasibility Trial:<\/b> The team conducted a mini-trial for 5 children with Progeria. The short, one-month \u201cfeasibility\u201d trial asked whether the three-drug combination would be well-tolerated, prior to embarking on a larger international trial. Side effects were acceptable, and the team moved ahead to the larger efficacy trial. \u00a0<\/b>\r\n\r\nThe Efficacy Trial:<\/b>\u00a045 children enrolled in this trial, from 24 different countries, speaking 17 different languages. This includes children that participated in the FTI-only trial, the 5 participants in the feasibility trial, and other children that were either too young to participate in the first trial or children that we discovered during the first clinical trial (after enrollment had ended). Children enrolled in the FTI-only trial had the opportunity to enroll in the triple trial when they participated in their last visit for the current trial. This allowed those children to continue taking FTI without any missed doses.\r\n\r\nThe Treatment\/Progeria Relationship <\/b>\r\n<\/b>How did we get from gene discovery to drug therapy for children with Progeria? Finding the gene for Progeria was the key. This gene is called LMNA, and it normally encodes a protein called prelamin A (this protein is further processed and becomes lamin A). Children with Progeria have a mutation in LMNA which leads to the production of an abnormal form of prelamin A called \u201cprogerin.\u201d Many years\u2019 worth of basic research on prelamin A and lamin A gave us the ability to understand that the drugs administered in this trial may affect disease in Progeria. Over the past six years, research has focused on systematically testing these drugs on Progeria cells and Progeria mice.\r\n\r\nThe Clinical Trial Team<\/b>\r\nSince the first trial began in May 2007, a 28-member team has treated children with Progeria from around the globe. Members of the team have expertise not only in Progeria, but also in the drugs administered.\r\n\r\nTrial Medications at a Glance<\/b>\r\nPravastatin<\/b> (marketed as Pravachol or Selektine) is a member of the drug class of statins. It is usually used for lowering cholesterol and preventing cardiovascular disease.\r\nZoledronic acid <\/b>is a bisphosphonate<\/b>, usually used as a bone drug for improving osteoporosis, and to prevent skeletal fractures in people suffering from some forms of cancer.\r\nLonafarnib<\/b> is an FTI <\/b>(Farnesyltransferase inhibitor), a drug that can reverse an abnormality in Progeria cells in the laboratory, and has improved disease in Progeria mice.\r\n\r\nAll 3 drugs block the production of the farnesyl molecule that is needed for progerin to create disease in Progeria. <\/i>\r\n<\/i>\r\n*<\/i><\/b> \u201c<\/b>Combined treatment with statins and aminobisphosphonates extends longevity in a mouse model of human premature aging\u201d<\/b>, by Ignacio Varela, Sandrine Pereira, Alejandro P. Ugalde, Claire L. Navarro, Mar\u00c4\u00b1a F. Suarez, Pierre Cau, Juan Cadinanos, Fernando G. Osorio, Nicolas Foray, Juan Cobo, Felix de Carlos, Nicolas Levy, Jose MP Freije and Carlos Lopez-Ot\u00c4\u00b1n. Nature Medicine, 2008. 14(7): p. 767-72.\r\n<\/a>\r\nThe new generation of children PRF is helping<\/b>\r\n![](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/AdaliaJAN09.jpg\")
\r\n\r\nIn March 2009, five children, ages 2-3, participated in the one-month feasibility study to determine if the side effects of the three drugs taken together were tolerable. The results were positive, paving the way for the full Triple Drug Trial to (initially) enroll up to 45 children with Progeria. Hats off to these amazing families! Here is what some of them had to say:\r\n<\/b>\r\n<\/b>\u201cEVERYONE has been so wonderful. To us you are ALL GOD SENT and we APPRECIATE all that you do for these little angels. Our family is so overwhelmed with excitement and all sorts of emotions with Adalia\u2019s trip to Boston this weekend, I can\u2019t even begin to type the words of how we are feeling.\"<\/em>\r\n\r\n\u201cThis new medication for Zach gives us a renewed hope that his heart will be stronger, his smile will be brighter and his life will be longer. This new drug trial is an answer to our prayers. Thank you to everyone involved with PRF who made this happen...the doctors, the researchers and the staff. You are our heroes!\u201d<\/em>\r\n![\"\"](\"https:\/\/www.progeriaresearch.org\/assets\/images\/news_images\/Cam-and-DadMarch09.jpg\")
Cam and his dad learn how to mix the FTI drug with a sweetener.<\/strong><\/span>[\/caption]\r\n\r\nThe \u201cTriple Trial\u201d was extended beyond its original 2-3-year timeframe, and expanded to include up to 80 children, so that every child could have access to treatment that may give them longer and healthier lives. However, during this last trial phase, all children are taking lonafarnib alone while the trial team analyzes the many thousands of data elements (each child underwent over 100 tests per visit!) on the three-drug therapy phase of the trial.\u00a0\u00a0 The triple therapy trial was pre-designed to include lonafarnib, pravastatin, and zoledronate.\u00a0 The hope is that adding two additional drugs to lonafarnib will boost the beneficial effects of the single drug therapy.\u00a0 While we know that lonafarnib is helping the children, it is still too early to tell if the other two are boosting the benefit.\u00a0 Usually, clinical trials run their course and the patients are taken off all the drugs until the data is analyzed to see if it helps; this could take years.\u00a0 PRF and the Boston Children\u2019s Hospital team made the important decision to add additional time of lonafarnib treatment<\/b> while the trial team explores other potential benefits and tracks any long-term side effects of lonafarnib, including lifespan. While the children continue to take the one known treatment, PRF and its research partners continue exploring additional treatment options - and have found one that they began testing in April 2016! Thus this last, lonafarnib only phase of the \u201cTriple Trial\u201d is overlapping with PRF\u2019s fourth trial, and all known children living with Progeria today have the opportunity to participate in a clinical trial.<\/a>\r\n\r\nWe are very happy with the visit\u2026To know that we will have a better quality of life for my baby makes me feel amazing, as my wife says he is a \"Warrior of Life \".<\/i>\r\n\r\nNew Drug, New Hope for Children with Progeria: Phase 1, 2-Drug Trial Began April 2016<\/strong>\r\n\r\nPRF is thrilled to announce that we are now funding and co-coordinating a new clinical trial, which will assess a two-drug combination of lonafarnib plus everolimus.\u00a0 Everolimus is a form of the drug rapamycin, <\/b>but everolimus can be more easily given to the children with Progeria because it requires fewer blood draws to measure drug levels.\u00a0 While lonafarnib may block progerin from developing, rapamycin appears to allow cells to more rapidly clear out the toxic progerin.\u00a0 Thus with rapamycin targeting a different pathway than lonafarnib, the combination may prove to be a \u201cone-two punch\u201d to Progeria - hopefully a better treatment than lonafarnib on its own.\r\n\r\nRapamycin<\/em> is an FDA-approved drug that has previously been shown to extend the lives of non-Progeria mouse models.\u00a0 A study* by researchers at the NIH in Bethesda, MD and Massachusetts General Hospital in Boston demonstrates that rapamycin decreases the amount of the disease-causing protein progerin by 50%, improves the abnormal nuclear shape, and extends the lifespan of Progeria cells in the laboratory.\r\n\r\nRapamycin is known for its anti-aging properties in mice. These findings are part of a growing list of studies that help to validate the theory that finding the cure for Progeria may also benefit the entire aging population.\r\n\r\n* K.\u00a0 Cao, J.\u00a0 J.\u00a0 Graziotto, C.\u00a0 D.\u00a0 Blair, J.\u00a0 R.\u00a0 Mazzulli, M.\u00a0 R.\u00a0 Erdos, D.\u00a0 Krainc, F.\u00a0 S.\u00a0 Collins, \u201cRapamycin Reverses\r\n\r\nCellular Phenotypes and Enhances Mutant Protein Clearance in Hutchinson-Gilford Progeria Syndrome Cells.\u201d Sci.\u00a0 Transl.\u00a0 Med.\u00a0 <\/em>3<\/strong>, 89ra58 (2011).\r\n\r\nPhase 1, to determine the dosage of everolimus, began in April 2016 and was successfully completed in June 2017, within the timeline and under the projected budget. The 17 children that participated in Phase 1 have been moved into Phase 2, which will test the effectiveness of the 2-drug combination. Enrollment of additional children began in July 2017. 2017 was a busy year, with 39 children from 18 countries traveling to Boston. Enrollment of additional children will continue through July 2018, and those already enrolled will be returning for their second visit. Together, this Phase 1-2 treatment trial may enroll up to 80 children, and take an estimated 3.5-4 years to complete, at a cost of $2.5 million dollars.\r\n\r\n![](\"https:\/\/www.progeriaresearch.org\/assets\/images\/medical_images\/Cao-rapamycin-image.jpg\")
The Progeria Research Foundation provided cells for this project from the PRF Cell & Tissue Bank<\/a> and helped fund the research through our grants program<\/a> \u2013 more proof that PRF\u2019s research-related programs are essential to advancements toward the cure.\r\n\r\nThis new trial is a collaborative effort that will build upon the knowledge gained from the previous Progeria trials. The children will be seen by virtually the same team of physicians from Boston Children\u2019s Hospital, Dana-Farber Cancer Institute and Brigham and Women\u2019s Hospital, all of whom now have world-renowned expertise in Progeria as well as the drugs involved.\r\n\r\nAs we begin this new chapter, PRF is and always has been responsible for\u00a0all trial expenses, including testing, travel, food, lodging, translators and staff. Each new trial is more expensive than the last, as more children enroll for a chance at longer, healthier lives. \u00a0 Please help us fund these vital trials \u2013 DONATE TODAY!<\/a>\r\n\r\n[caption id=\"attachment_3791\" align=\"aligncenter\" width=\"250\"]![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/02\/Ana-Clara-trial-page.jpg\")
8-year-old Ana Clara shares a special moment with her mother while taking a break from testing at Boston Children\u2019s Hospital. She recently enrolled in phase 2 of the Progeria clinical trial. Travel from her remote town in Brazil included a 10-hour bus ride to the airport.<\/span><\/strong><\/span>[\/caption]\r\n\r\n[\/vc_column_text][\/vc_column][\/vc_row][vc_row][vc_column][\/vc_column][\/vc_row][vc_row][vc_column][vc_column_text]\r\n\r\n[\/vc_column_text][\/vc_column][\/vc_row]\t\t","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-772","page","type-page","status-publish","hentry"],"yoast_head":"\n