Italiano: [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 global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55\u2033 background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/About-Header.jpg\u201d background_position=\u201dcenter_left\u201d custom_padding=\u201d9vw||9vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited="on|desktop" title_font_size_tablet="45px" title_font_size_phone="40px" title_font_size_last_edited="on|phone" z_index_tablet="500" custom_css_main_element="background-position: center 18% !important;" global_colors_info="{}"]<\/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=\u201dhandprint-bg\u201d _builder_version=\u201d4.16\u2033 background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/blue-handprint-only.png\u201d parallax=\u201don\u201d parallax_method=\u201doff\u201d larghezza_interna=\u201d100%\u201d larghezza_interna_massima=\u201d100%\u201d imbottitura_personalizzata=\u201d0|0px|54px|0px|falso|falso\u201d z_index_tablet=\u201d500\u2033 larghezza_interna_massima=\u201d10px\u201d colore_interna_massima=\u201d#8fd2ed\u201d usa_larghezza_personalizzata=\u201don\u201d unit\u00e0_di_larghezza=\u201doff\u201d percentuale_larghezza_personalizzata=\u201d100%\u201d info_colori_globali=\u201d{}\u201d][et_pb_column tipo=\u201d1_4\u2033 versione_builder=\u201d4.16\u2033 imbottitura_personalizzata=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_sidebar area=\u201det_pb_widget_area_18\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 global_colors_info=\u201d{}\u201d]
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L'inibitore della farnesiltransferasi (FTI) lonafarnib (commercializzato come Zokinvy) \u00e8 il primo e unico trattamento farmacologico noto per i bambini affetti da Progeria.<\/p>\n
La storia dietro questa scoperta storica: nell'agosto 2005 e nel febbraio 2006, i ricercatori hanno pubblicato studi che supportavano un potenziale trattamento farmacologico per i bambini affetti da Progeria. Originariamente sviluppato per trattare potenzialmente il cancro, il lonafarnib ha invertito le drammatiche anomalie della struttura nucleare che sono il segno distintivo delle cellule dei bambini affetti da Progeria. Inoltre, questo farmaco FTI ha migliorato alcuni segni della malattia in un modello murino simile alla Progeria.<\/p>\n
Perch\u00e9 i ricercatori hanno pensato che questo farmaco avrebbe funzionato nella Progeria? La proteina che riteniamo responsabile della Progeria \u00e8 chiamata progerina. Per bloccare la normale funzione cellulare e causare la Progeria, una molecola chiamata "gruppo farnesile" deve essere attaccata alla proteina progerina. Gli FTI agiscono bloccando (inibendo) l'attaccamento del gruppo farnesile sulla progerina. Quindi, se il farmaco FTI pu\u00f2 bloccare questo attaccamento del gruppo farnesile nei bambini con Progeria, allora la progerina potrebbe essere "paralizzata" e la Progeria potrebbe migliorare.<\/p>\n
Per la prima volta, avevamo davanti a noi un possibile trattamento per i bambini affetti da Progeria. primo studio clinico sul farmaco Progeria<\/a> \u00e8 iniziato nel 2007 e nel 2012 sono stati pubblicati i risultati dello studio, dimostrando che ogni bambino ha sperimentato miglioramenti in una o pi\u00f9 aree, incluso il vitale sistema cardiovascolare. Nel maggio 2014, un ulteriore studio ha rivelato che il lonafarnib aumenta la durata di vita stimata di almeno 1,6 anni (che sarebbe poi aumentata, con l'ulteriore avanzamento dello studio) e nell'aprile 2018 uno studio pubblicato in Il Journal dell'American Medical Association (JAMA)<\/em> hanno riferito che il lonafarnib da solo prolungava la sopravvivenza nei bambini affetti da Progeria. \u00a0clicca qui<\/a> per i dettagli sullo studio del 2012,\u00a0Qui <\/a>per i dettagli sui risultati del 2014 e Qui<\/a> per i dettagli sullo studio del 2018.<\/p>\n Nel maggio 2018, sulla scia dello studio pubblicato in GIORNO<\/em>, PRF ed Eiger Biopharmaceuticals hanno stipulato un accordo di collaborazione e fornitura per lo sviluppo e la revisione da parte della Food and Drug Administration statunitense e la potenziale approvazione del lonafarnib per il trattamento della Progeria nei bambini. Il deposito \u00e8 stato completato il 23 marzo 2020. <\/a>Nel novembre 2020, il lonafarnib \u00e8 entrato nella storia come il primo trattamento in assoluto a ricevere l'approvazione della Food and Drug Administration (FDA) statunitense<\/a> per la Progeria e le Laminopatie Progeroidi.<\/p>\n Attualmente disponiamo di una cura per la Progeria, ma non \u00e8 una cura definitiva. Continuiamo quindi a portare avanti la ricerca per scoprire farmaci ancora pi\u00f9 efficaci e, in ultima analisi, in grado di curare la Progeria.<\/p>\n Italiano: [\/et_pb_text][\/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=\u201d4.3.4\u2033 background_color=\u201d#29327a\u201d custom_margin=\u201d25px|||||\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 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=\u201dIscriviti ora\u201d admin_label=\u201dIscriviti alla 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="on|25px|25px|25px|25px" global_colors_info="{}" button_bg_color__hover_enabled="on" button_bg_color__hover="#8fd2ed" button_border_color__hover_enabled="on"]<\/p>\n [\/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=\u201dFai una donazione ora\u201d admin_label=\u201dInsieme troveremo la cura!\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 colore_bg_pulsante__hover=\u201d#8fd2ed\u201d colore_bordo_pulsante__hover_abilitato=\u201don\u201d]<\/p>\n [\/et_pb_cta][\/et_pb_column][et_pb_column tipo=\u201d1_2\u2033 _builder_version=\u201d4.16\u2033 imbottitura_personalizzata=\u201d|||\u201d global_colors_info=\u201d{}\u201d custom_padding__hover=\u201d|||\u201d][et_pb_image src=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2021\/06\/June2021hp4temppsd.png\u201d title_text=\u201dJune2021hp4temppsd\u201d _builder_version=\u201d4.16\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":" Italiano: [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 global_colors_info=\u201d{}\u201d][et_pb_fullwidth_header _builder_version=\u201d4.16\u2033 title_font_size=\u201d55\u2033 background_color=\u201d#29327a\u201d background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/About-Header.jpg\u201d background_position=\u201dcenter_left\u201d custom_padding=\u201d9vw||9vw||true\u201d custom_padding_tablet=\u201d\u201d custom_padding_phone=\u201d|56px||\u201d custom_padding_last_edited="on|desktop" title_font_size_tablet="45px" title_font_size_phone="40px" title_font_size_last_edited="on|phone" z_index_tablet="500" custom_css_main_element="background-position: center 18% !important;" global_colors_info=\u201d{}\u201d] Il farmaco FTI [\/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=\u201dhandprint-bg\u201d _builder_version=\u201d4.16\u2033 background_image=\u201dhttps:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/blue-handprint-only.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 [\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=\"The FTI Drug Lonafarnib\" font_container=\"tag:h1|text_align:center\" use_theme_fonts=\"yes\"][vc_custom_heading text=\"The FTI lonafarnib \u2013 the first and only known drug treatment for children with Progeria\" use_theme_fonts=\"yes\"][vc_column_text]In August 2005 and February 2006, researchers published studies that supported a potential drug treatment for children with Progeria. Farnesyltransferase inhibitors (FTIs)<\/b>, originally developed to potentially treat cancer, reversed the dramatic nuclear structure abnormalities that are the hallmark of cells from children with Progeria. In addition, this FTI drug improves some signs of disease in a Progeria-like mouse model.\r\n\r\nWhy did researchers think this drug would work in Progeria?<\/b> The 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.\r\n\r\nFor the first time, we had in front of us a possible treatment for children with Progeria.\u00a0<\/b>A first-ever clinical drug trial<\/b><\/a>\u00a0began in 2007, and in 2012 <\/b>the study results were published, demonstrating that every child experienced improvement in one or more areas, including the vital cardiovascular system.\u00a0 In May 2014, further study revealed lonafarnib 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)\u00a0and in April 2018 a study published in The Journal of the American Medical Association (JAMA)<\/em> reported that lonafarnib alone extended survival in children with Progeria. \u00a0Click here<\/b><\/a> for details on the 2012 study,\u00a0here<\/b> <\/a>for details on the 2014 findings, and here<\/a><\/strong> for details on the 2018 study.\r\n\r\nIn May 2018, on the heels of the study published in JAMA<\/em>, PRF and Eiger Biopharmaceuticals entered into collaboration and supply agreement for the development and pursuit of U.S. Food and Drug Administration review and potential approval of lonafarnib for the treatment of Progeria in children. Lonafarnib will mark the first therapy submitted to the FDA for the treatment of Progeria.\r\n\r\nWe now have one treatment for Progeria but it is not a cure, so we continue to drive the research to discover drugs that will be even more effective, and ultimately cure Progeria.[\/vc_column_text][\/vc_column][\/vc_row]\t\t","_et_gb_content_width":"","footnotes":"","_links_to":"","_links_to_target":""},"class_list":["post-796","page","type-page","status-publish","hentry"],"yoast_head":"\nIscrizione<\/h2>\n
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