[et_pb_section fb_built=”1″ fullwidth=”on” disabled_on=”off|off|off” _builder_version=”4.16″ border_width_bottom=”55px” border_color_bottom=”#29327a” locked=”off” global_colors_info=”{}”][et_pb_fullwidth_header _builder_version=”4.16″ title_font_size=”55px” background_color=”#29327a” background_image=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/09\/Kids-on-Bridge.png” custom_padding=”11.5vw||11.5vw||true” custom_padding_tablet=”” custom_padding_phone=”|56px||” 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;” title_text_shadow_horizontal_length_tablet=”0px” title_text_shadow_vertical_length_tablet=”0px” title_text_shadow_blur_strength_tablet=”1px” content_text_shadow_horizontal_length_tablet=”0px” content_text_shadow_vertical_length_tablet=”0px” content_text_shadow_blur_strength_tablet=”1px” subhead_text_shadow_horizontal_length_tablet=”0px” subhead_text_shadow_vertical_length_tablet=”0px” subhead_text_shadow_blur_strength_tablet=”1px” content_link_text_shadow_horizontal_length_tablet=”0px” content_link_text_shadow_vertical_length_tablet=”0px” content_link_text_shadow_blur_strength_tablet=”1px” content_ul_text_shadow_horizontal_length_tablet=”0px” content_ul_text_shadow_vertical_length_tablet=”0px” content_ul_text_shadow_blur_strength_tablet=”1px” content_ol_text_shadow_horizontal_length_tablet=”0px” content_ol_text_shadow_vertical_length_tablet=”0px” content_ol_text_shadow_blur_strength_tablet=”1px” content_quote_text_shadow_horizontal_length_tablet=”0px” content_quote_text_shadow_vertical_length_tablet=”0px” content_quote_text_shadow_blur_strength_tablet=”1px” button_one_text_shadow_horizontal_length_tablet=”0px” button_one_text_shadow_vertical_length_tablet=”0px” button_one_text_shadow_blur_strength_tablet=”1px” button_two_text_shadow_horizontal_length_tablet=”0px” button_two_text_shadow_vertical_length_tablet=”0px” button_two_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” box_shadow_horizontal_image_tablet=”0px” box_shadow_vertical_image_tablet=”0px” box_shadow_blur_image_tablet=”40px” box_shadow_spread_image_tablet=”0px” box_shadow_horizontal_button_one_tablet=”0px” box_shadow_vertical_button_one_tablet=”0px” box_shadow_blur_button_one_tablet=”40px” box_shadow_spread_button_one_tablet=”0px” box_shadow_horizontal_button_two_tablet=”0px” box_shadow_vertical_button_two_tablet=”0px” box_shadow_blur_button_two_tablet=”40px” box_shadow_spread_button_two_tablet=”0px” text_shadow_horizontal_length_tablet=”0px” text_shadow_vertical_length_tablet=”0px” text_shadow_blur_strength_tablet=”1px” global_colors_info=”{}”]<\/p>\n
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\n[\/et_pb_sidebar][\/et_pb_column][et_pb_column type=”3_4″ specialty_columns=”3″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|phone” disabled_on=”on|on|” _builder_version=”4.16″ custom_padding=”|35px||0px” custom_padding_tablet=”|35px||35px||true” custom_padding_phone=”” animation_direction=”top” z_index_tablet=”500″ global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Progeria Clinical Drug Trials: Background” _builder_version=”4.27.2″ header_4_line_height=”1.2em” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ text_text_shadow_horizontal_length_tablet=”0px” text_text_shadow_vertical_length_tablet=”0px” text_text_shadow_blur_strength_tablet=”1px” link_text_shadow_horizontal_length_tablet=”0px” link_text_shadow_vertical_length_tablet=”0px” link_text_shadow_blur_strength_tablet=”1px” ul_text_shadow_horizontal_length_tablet=”0px” ul_text_shadow_vertical_length_tablet=”0px” ul_text_shadow_blur_strength_tablet=”1px” ol_text_shadow_horizontal_length_tablet=”0px” ol_text_shadow_vertical_length_tablet=”0px” ol_text_shadow_blur_strength_tablet=”1px” quote_text_shadow_horizontal_length_tablet=”0px” quote_text_shadow_vertical_length_tablet=”0px” quote_text_shadow_blur_strength_tablet=”1px” header_text_shadow_horizontal_length_tablet=”0px” header_text_shadow_vertical_length_tablet=”0px” header_text_shadow_blur_strength_tablet=”1px” header_2_text_shadow_horizontal_length_tablet=”0px” header_2_text_shadow_vertical_length_tablet=”0px” header_2_text_shadow_blur_strength_tablet=”1px” header_3_text_shadow_horizontal_length_tablet=”0px” header_3_text_shadow_vertical_length_tablet=”0px” header_3_text_shadow_blur_strength_tablet=”1px” header_4_text_shadow_horizontal_length_tablet=”0px” header_4_text_shadow_vertical_length_tablet=”0px” header_4_text_shadow_blur_strength_tablet=”1px” header_5_text_shadow_horizontal_length_tablet=”0px” header_5_text_shadow_vertical_length_tablet=”0px” header_5_text_shadow_blur_strength_tablet=”1px” header_6_text_shadow_horizontal_length_tablet=”0px” header_6_text_shadow_vertical_length_tablet=”0px” header_6_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”]<\/p>\n
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Progeria klinische medicijnproeven zijn de beste hoop voor kinderen met Progeria, waarbij mogelijke behandelingen worden getest die hen in staat kunnen stellen om langer en gezonder te leven. Deze proeven zijn het hoogtepunt van jarenlang onderzoek gericht op welk medicijn of welke combinatie van medicijnen de kinderen zal behandelen en genezen.<\/p>\n
Sinds 1999, toen we PRF oprichtten en er geen middelen waren voor deze kinderen, zijn we van totale onbekendheid naar genenonderzoek gegaan, naar de eerste klinische proeven met Progeria, naar de allereerste door de FDA goedgekeurde behandeling, genaamd lonafarnib \u2013 allemaal in een tempo dat vrijwel ongehoord is in de wetenschappelijke gemeenschap. En terwijl we deze handvol kinderen helpen, heeft de connectie van Progeria met veelvoorkomende hartziekten en veroudering enorme implicaties voor ons allemaal.<\/p>\n
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<\/strong><\/em><\/strong><\/h4>\n<\/em><\/strong><\/p>\n PRF is, in samenwerking met de in Korea gevestigde sponsor van de studie PRG Science & Technology (PRG S&T), begonnen met de inschrijving voor een gloednieuwe klinische proef met een medicijn genaamd Progerinin. Laboratoriumbewijs toont aan dat dit medicijn, in combinatie met lonafarnib, effectiever kan zijn dan lonafarnib alleen. PRF financierde het laboratoriumwerk dat leidde tot de oprichting van PRG S&T en de ontwikkeling van Progerinin. Het vooronderzoek is begonnen in het Boston Children's Hospital, in afwachting van het aantrekken van kinderen van over de hele wereld om zich in de komende maanden in te schrijven voor deze proef. We zijn superenthousiast over het starten van een nieuwe proef met zo'n veelbelovend medicijn en kijken ernaar uit om meer details met u te delen zodra deze beschikbaar zijn.<\/p>\n Neem contact op met Shelby Phillips voor meer informatie over het proces. PRF-medeoprichter en directeur Audrey Gordon sluit in juni de deal met Dr. Bum-Joon Park van PRG S&T om door te gaan met het voorbereidende werk.<\/em><\/p>\n <\/em><\/p>\n [\/et_pb_text][et_pb_text admin_label=”RNA Therapy: Drug Administration Feasibility Study is Complete!” _builder_version=”4.27.2″ header_4_line_height=”1.2em” vertical_offset_tablet=”0″ horizontal_offset_tablet=”0″ custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ text_text_shadow_horizontal_length_tablet=”0px” text_text_shadow_vertical_length_tablet=”0px” text_text_shadow_blur_strength_tablet=”1px” link_text_shadow_horizontal_length_tablet=”0px” link_text_shadow_vertical_length_tablet=”0px” link_text_shadow_blur_strength_tablet=”1px” ul_text_shadow_horizontal_length_tablet=”0px” ul_text_shadow_vertical_length_tablet=”0px” ul_text_shadow_blur_strength_tablet=”1px” ol_text_shadow_horizontal_length_tablet=”0px” ol_text_shadow_vertical_length_tablet=”0px” ol_text_shadow_blur_strength_tablet=”1px” quote_text_shadow_horizontal_length_tablet=”0px” quote_text_shadow_vertical_length_tablet=”0px” quote_text_shadow_blur_strength_tablet=”1px” header_text_shadow_horizontal_length_tablet=”0px” header_text_shadow_vertical_length_tablet=”0px” header_text_shadow_blur_strength_tablet=”1px” header_2_text_shadow_horizontal_length_tablet=”0px” header_2_text_shadow_vertical_length_tablet=”0px” header_2_text_shadow_blur_strength_tablet=”1px” header_3_text_shadow_horizontal_length_tablet=”0px” header_3_text_shadow_vertical_length_tablet=”0px” header_3_text_shadow_blur_strength_tablet=”1px” header_4_text_shadow_horizontal_length_tablet=”0px” header_4_text_shadow_vertical_length_tablet=”0px” header_4_text_shadow_blur_strength_tablet=”1px” header_5_text_shadow_horizontal_length_tablet=”0px” header_5_text_shadow_vertical_length_tablet=”0px” header_5_text_shadow_blur_strength_tablet=”1px” header_6_text_shadow_horizontal_length_tablet=”0px” header_6_text_shadow_vertical_length_tablet=”0px” header_6_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”]<\/p>\n PRF heeft de eerste pati\u00ebntbetrokken stappen gezet richting een klinische proef met RNA-therapie \u2013 ZO spannend!<\/p>\n Achtergrond: In januari 2021 rapporteerden we baanbrekende bevindingen in RNA-therapie<\/strong>, waarbij deze therapie geremde productie van RNA dat codeert voor het ziekteveroorzakende eiwit progerine, Progeria<\/strong>. Het onderzoek*, geleid door Dr. Francis Collins, wetenschappelijk adviseur van het Witte Huis en voormalig directeur van de National Institutes of Health (NIH), onthulde dat Progeria-muizen die behandeld werden met een medicijn genaamd SRP-2001 verminderde de schadelijke progerine mRNA- en prote\u00efne-expressie in bloedvaten<\/strong>, en ook in andere weefsels. De bloedvaten waren sterker en de muizen vertoonden een verhoogde overleving van meer dan 60%<\/strong> vergeleken met onbehandelde muizen. Zo werd het werk met deze veelbelovende therapie voortgezet en hebben we de\u00a0volgende stap <\/strong>met een Haalbaarheidsstudie <\/strong>als volgt:<\/p>\n Meestal zijn RNA-therapeutica vloeistoffen die intraveneus (direct in de ader) worden ge\u00efnjecteerd. Mensen met Progeria zouden echter de intraveneuze toediening van de vereiste dagelijkse dosering niet kunnen verdragen. Daarom ontwikkelde PRF een subcutaan toedieningssysteem<\/strong> waarbij de vloeistof met een kleine naald onder de huid kan worden ge\u00efnjecteerd. Er werd een 6 maanden durend onderzoek bij BCH afgerond om de haalbaarheid van deze leveringsmethode voor mensen met Progeria te bepalen<\/strong>. Het team testte of toediening van een zoutoplossing comfortabel subcutaan kan worden ge\u00efnjecteerd en welk injectiesysteem en welke injectieplaats het meest comfortabel zouden zijn. Zodra de gegevens van deze studie zijn geanalyseerd, zullen we een stap dichter bij een klinische proef in gentherapie<\/strong>!<\/p>\n *<\/strong>Erdos, MR, Cabral, WA, Tavarez, UL et al. Een gerichte antisense therapeutische benadering voor Hutchinson-Gilford progeriasyndroom. Nat Med<\/em> (2021).<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”What\u2019s Next for Treatments and Cure? ” _builder_version=”4.27.0″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” custom_margin=”|||15px|false|false” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”What\u2019s Happening TODAY With the Progeria Clinical Trials? ” _builder_version=”4.27.0″ header_4_line_height=”1.2em” vertical_offset_tablet=”0″ horizontal_offset_tablet=”0″ custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ text_text_shadow_horizontal_length_tablet=”0px” text_text_shadow_vertical_length_tablet=”0px” text_text_shadow_blur_strength_tablet=”1px” link_text_shadow_horizontal_length_tablet=”0px” link_text_shadow_vertical_length_tablet=”0px” link_text_shadow_blur_strength_tablet=”1px” ul_text_shadow_horizontal_length_tablet=”0px” ul_text_shadow_vertical_length_tablet=”0px” ul_text_shadow_blur_strength_tablet=”1px” ol_text_shadow_horizontal_length_tablet=”0px” ol_text_shadow_vertical_length_tablet=”0px” ol_text_shadow_blur_strength_tablet=”1px” quote_text_shadow_horizontal_length_tablet=”0px” quote_text_shadow_vertical_length_tablet=”0px” quote_text_shadow_blur_strength_tablet=”1px” header_text_shadow_horizontal_length_tablet=”0px” header_text_shadow_vertical_length_tablet=”0px” header_text_shadow_blur_strength_tablet=”1px” header_2_text_shadow_horizontal_length_tablet=”0px” header_2_text_shadow_vertical_length_tablet=”0px” header_2_text_shadow_blur_strength_tablet=”1px” header_3_text_shadow_horizontal_length_tablet=”0px” header_3_text_shadow_vertical_length_tablet=”0px” header_3_text_shadow_blur_strength_tablet=”1px” header_4_text_shadow_horizontal_length_tablet=”0px” header_4_text_shadow_vertical_length_tablet=”0px” header_4_text_shadow_blur_strength_tablet=”1px” header_5_text_shadow_horizontal_length_tablet=”0px” header_5_text_shadow_vertical_length_tablet=”0px” header_5_text_shadow_blur_strength_tablet=”1px” header_6_text_shadow_horizontal_length_tablet=”0px” header_6_text_shadow_vertical_length_tablet=”0px” header_6_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”]<\/p>\n <\/p>\n Het meest recente onderzoek omvatte 2 medicijnen: lonafarnib<\/strong>\u00a0en een nieuw medicijn, everolimus<\/strong>Fase 1, om de veilige en geschikte dosering van everolimus te bepalen, begon in april 2016 en werd succesvol afgerond in juni 2017. Fase 2, waarin de effectiviteit van de combinatie van 2 geneesmiddelen werd getest, begon in juli 2017 en werd afgerond in april 2022. Zestig kinderen uit 27 landen namen deel aan deze fase met twee geneesmiddelen!<\/p>\n Everolimus is een vorm van het medicijn rapamycine; everolimus kan gemakkelijker worden gegeven aan kinderen met Progeria omdat er minder bloed hoeft te worden afgenomen om de medicijnspiegels te meten. Terwijl lonafarnib de ontwikkeling van het giftige progerine kan blokkeren, lijkt rapamycine cellen in staat te stellen progerine sneller op te ruimen. Dus met rapamycine gericht op een ander pad dan lonafarnib, kan de combinatie een "one-two punch" blijken te zijn voor Progeria - hopelijk een betere behandeling dan lonafarnib op zichzelf.<\/p>\n We zijn nu een periode van data-analyse ingegaan en verwachten uiteindelijk de resultaten te publiceren in een peer-reviewed, wetenschappelijk tijdschrift. In de tussentijd zijn de deelnemers aan de trial overgegaan naar de monotherapie-extensie van de trial, of naar een ander toegangsprogramma. Via beide trajecten blijven deelnemers lonafarnib krijgen, de huidige standaardzorg.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Trials History at a Glance” _builder_version=”4.27.0″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” custom_margin=”|||15px|false|false” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|phone” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.22.1″ custom_margin=”0px||||false|false” custom_padding=”|35px|0px|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”To date, PRF has funded and co-coordinated three clinical trials” _builder_version=”4.27.0″ text_font=”||||||||” header_4_font=”||||||||” header_4_line_height=”1.2em” custom_padding=”||0px|||” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” global_colors_info=”{}”]<\/p>\n Tot op heden heeft PRF vier klinische onderzoeken gefinancierd en geco\u00f6rdineerd<\/strong>. PRF is en is altijd verantwoordelijk geweest voor alle kosten van de proef, inclusief testen, reizen, eten, onderdak, vertalers en personeel. Elke nieuwe proef is duurder dan de vorige, omdat meer kinderen zich inschrijven voor een kans op een langer, gezonder leven.<\/p>\n [\/et_pb_text][et_pb_button button_url=”https:\/\/progeriaresearch.donorsupport.co\/-\/XZHJVWZR” url_new_window=”on” button_text=”Please help us fund these vital trials. Donate Today” admin_label=”Donate” _builder_version=”4.22.1″ background_layout=”dark” custom_margin=”20px||25px” animation_style=”slide” animation_direction=”left” animation_intensity_slide=”25%” global_colors_info=”{}”] #1 omvatte \u00e9\u00e9n medicijn, lonafarnib, begon in 2007 en bleek succesvol. Lees alles over de historische behandelingsontdekking hier.<\/a><\/p>\n #2, het haalbaarheidsgedeelte van de "Triple Trial" omvatte 3 medicijnen: lonafarnib, pravastatine en zoledronaat. Deze 1 maand durende, fase 1 "mini-trial" vond plaats in maart 2009 om te bepalen of het toevoegen van 2 extra medicijnen aan het lonafarnib-regime veilig was om door te gaan met een grotere populatie (wat het geval was).<\/p>\n #3, de \u201cTriple Trial,\u201d was een voortzetting van de Feasibility trial, en was een fase 2-studie, die begon in augustus 2009. Het protocol veranderde in de loop van vijf jaar, waarbij werd teruggegaan naar alleen lonafarnib en de inschrijving werd heropend, zodat meer kinderen konden deelnemen. Lees meer hier<\/a>.<\/p>\n #4 is de proef met twee geneesmiddelen, lonafarnib en everolimus. Fase 1, om de veilige en geschikte dosering van everolimus te bepalen, begon in april 2016 en werd succesvol afgerond in juni 2017. Fase 2, die de effectiviteit van de combinatie van twee geneesmiddelen testte, begon in juli 2017 en werd afgerond in april 2022. De monotherapie-uitbreiding van deze proef gaat vandaag verder.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|desktop” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2018\/02\/Ana-Clara-trial-page.jpg” align=”center” align_tablet=”center” align_phone=”” align_last_edited=”on|desktop” admin_label=”liv” _builder_version=”4.22.1″ border_width_all=”4px” border_color_all=”#392887″ global_colors_info=”{}”] In 2006 identificeerden onderzoekers een mogelijke medicamenteuze behandeling voor kinderen met Progeria,\u00a0zogenaamde FTI's.<\/a>\u00a0Voor het eerst kregen we een mogelijke behandeling voor kinderen met Progeria voor ogen.\u00a0<\/b>Spannende tijden! De klinische proef met Progeria begon op 7 mei 2007 met twee kinderen \u2013 Meghan en Megan \u2013 die aankwamen bij Boston Children's Hospital in Boston, MA voor hun eerste van zeven bezoeken in een periode van 2 jaar. Bij dit eerste bezoek werden ze uitgebreid getest en kregen ze hun eerste doses van het medicijn. Gemiddeld reisden er daarna elke week twee gezinnen naar Boston, tot en met december 2009, gevolgd door een periode waarin het proefteam de vele duizenden data-elementen analyseerde (elk kind onderging meer dan 100 testen per bezoek!) en de publicatie van de resultaten nastreefde.<\/p>\n <\/p>\n [\/et_pb_text][et_pb_testimonial author=”Francis Collins, MD, PhD” job_title=”Director of the National Human Genome Research Institutethat mapped the human genome, workshop speaker and co-discoverer of the Progeria gene.” quote_icon_color=”#00b2e2″ portrait_width=”140px” portrait_height=”140px” _builder_version=”4.16″ background_color=”#f7f7f7″ custom_margin_tablet=”” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” z_index_tablet=”500″ custom_css_testimonial_author=”color: #00b2e2;||font-size: 19px !important;||font-weight: 700;” custom_css_testimonial_meta=”font-style: italic;||font-weight: 700;” box_shadow_style_image=”preset3″ locked=”off” global_colors_info=”{}”]<\/p>\n \u201cIk ken geen enkele andere zeldzame genetische ziekte die in minder dan vier jaar van genontdekking naar klinische proef is gegaan \u2013 een fenomenaal bewijs van het harde werk van The Progeria Research Foundation.\u201d<\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”1_2,1_2″ custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”false” padding_left_right_link_2=”false” _builder_version=”4.16″ custom_margin=”||25px” custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|20px||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/11\/redoof2007photocollage.png” title_text=”redoof2007photocollage” align_tablet=”center” align_phone=”” align_last_edited=”on|desktop” admin_label=”collage” _builder_version=”4.27.0″ custom_margin=”|-50px|||false|false” global_colors_info=”{}”] <\/p>\n Achtentwintig (28) kinderen uit zestien landen namen deel, in de leeftijd van 3 tot 15 jaar. Kinderen keerden elke vier maanden terug naar het Children's Hospital Boston om te testen en om nieuwe medicijnen te krijgen, en bleven bij elk bezoek 4-8 dagen in Boston. Terwijl ze thuis waren, hielden hun artsen de kinderen nauwlettend in de gaten en dienden ze periodieke gezondheidsrapporten in bij het onderzoeksteam van Boston. Gedurende de duur van het onderzoek reisden gemiddeld 2 kinderen per week naar Boston om deel te nemen.<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Who, Where, When, How and How Much\u2026” _builder_version=”4.27.0″ header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”The first three clinical trials were led by Mark Kieran MD” _builder_version=”4.27.0″ header_4_line_height=”1.2em” custom_margin=”||25px” custom_margin_tablet=”35px||” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n De eerste drie klinische onderzoeken werden geleid door Mark Kieran MD, PhD,\u00a0<\/strong>Directeur, Pediatric Medical Neuro-Oncology, Dana-Farber Cancer Institute en Children's Hospital Boston; universitair docent, afdelingen Pediatrie en Hematologie\/Oncologie, Harvard Medical School. Dr. Kieran is een pediatrische oncoloog met uitgebreide ervaring met het onderzochte geneesmiddel (farnesyltransferase of FTI) bij kinderen. In 2017 verliet hij zijn functie bij Dana Farber om in de particuliere sector te werken. Co-voorzitters waren Monica Kleinman, MD, directeur van de Medical-Surgical Intensive Care Unit, Sr. Associate in Critical Care Medicine bij BCH, universitair docent aan Harvard Medical School; en Leslie Gordon, MD, PhD, medisch directeur van PRF, docent aan BCH en Harvard Medical School, hoogleraar Pediatrie aan Hasbro Children's Hospital en Brown University in Providence, RI. Dr. Kleinman heeft de leidende rol van Principal Investigator op zich genomen.<\/p>\n De klinische proeven zijn een gezamenlijke inspanning van artsen van het Boston Children's Hospital, het Dana-Farber Cancer Institute en het Brigham and Women's Hospital.,<\/strong>\u00a0alle Harvard University-instellingen. Daarnaast hielpen artsen en wetenschappers van The Warren Alpert Medical School aan Brown University en NIH om deze eerste en de andere proeven tot een succes te maken.<\/p>\n [\/et_pb_text][et_pb_text admin_label=”How did we get to this point?” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n In 2003 heeft het gezamenlijke onderzoeksteam van de Progeria Research Foundation\u00a0\u00a0ontdekte het Progeria-gen<\/a>.<\/a> Deze ontdekking leidde niet alleen tot meer begrip van Progeria, maar wetenschappers weten nu dat het bestuderen van Progeria ons kan helpen meer te leren over hartziekten en het normale verouderingsproces dat ons allemaal treft. Sinds de ontdekking van het gen heeft de steun van onderzoekers, clinici, families van kinderen met Progeria en mensen zoals JIJ ons naar een ander kruispunt gebracht in de zoektocht naar een behandeling. Onderzoekers zijn begonnen met een intensieve studie van dit vijandige eiwit genaamd\u00a0progerine<\/em>, en in 2006 identificeerden ze een potenti\u00eble medicijnbehandeling voor kinderen met Progeria, genaamd farnesyltransferase-remmers (FTI's), en voerden ze onderzoeken uit in het lab die een menselijke proef met het medicijn ondersteunden. De gekozen FTI werd aanvankelijk geleverd door Merck en werd lonafarnib<\/em>.\u00a0Klik hier<\/a>\u00a0voor meer details over het onderzoek.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Why did researchers think this drug would work in Progeria?” _builder_version=”4.16″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Normale cel, Progeria cel, Progeria cel na behandeling met FTI.<\/p><\/div>\n Het eiwit waarvan wij denken dat het verantwoordelijk is voor Progeria heet progerine. Om de normale celfunctie te blokkeren en Progeria te veroorzaken, moet een molecuul genaamd een "farnesylgroep" aan het progerine-eiwit worden gehecht. FTI's werken door de hechting van de farnesylgroep aan progerine te blokkeren (remmen). Dus als het FTI-medicijn deze farnesylgroephechting bij kinderen met Progeria kan blokkeren, dan kan progerine "verlamd" worden en Progeria verbeterd.\u00a0\u00a0Klik hier<\/a>\u00a0voor meer informatie over FTI's.<\/p>\n [\/et_pb_text][et_pb_text admin_label=”How did PRF fund the trial?” _builder_version=”4.16″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Dankzij de steun van duizenden konden we alle benodigde fondsen werven om de kosten van het proces te dekken. Onze oprechte dank gaat uit naar iedereen die zijn "tijd, talenten en geld" heeft bijgedragen om deze ongelooflijke prestatie mogelijk te maken, en natuurlijk naar alle moedige families die hebben deelgenomen.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|35px|35.2344px|35px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”The FTI lonafarnib is now a proven treatment for Progeria.” _builder_version=”4.22.1″ text_font=”||||||||” header_font=”||||||||” header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” global_colors_info=”{}”]<\/p>\n <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”2012 2014″ _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”39px||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n In 2012 werden de onderzoeksresultaten gepubliceerd, waaruit bleek dat elk kind verbetering ervoer op een of meer gebieden, waaronder het vitale cardiovasculaire systeem. In mei 2014 toonde een onderzoek aan dat een of meer van de 3 geneesmiddelen \u2013 waaronder lonafarnib \u2013 die werden getest in door PRF gefinancierde klinische onderzoeken, de levensduur verlengden; het was onduidelijk welk geneesmiddel deze positieve, levensveranderende impact had. In april 2018 werd echter een onderzoek gepubliceerd in Het tijdschrift van de American Medical Association (JAMA)<\/em> meldden dat lonafarnib alleen de overleving van kinderen met Progeria met ten minste 1,6 jaar verlengde. Klik hier<\/a>\u00a0<\/a>voor details over de historische behandelingsontdekkingsstudie uit 2012,\u00a0hier\u00a0<\/a>voor details over de bevindingen van 2014, en\u00a0hier<\/a>\u00a0voor meer informatie over het onderzoek uit 2018.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_testimonial portrait_url=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cam-and-DadMarch09.jpg” quote_icon_color=”#00b2e2″ portrait_width=”140px” portrait_height=”140px” _builder_version=”4.16″ background_color=”#f7f7f7″ custom_margin_tablet=”” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” z_index_tablet=”500″ custom_css_testimonial_author=”color: #00b2e2;||font-size: 19px !important;||font-weight: 700;” custom_css_testimonial_meta=”font-style: italic;||font-weight: 700;” box_shadow_style_image=”preset3″ locked=”off” global_colors_info=”{}”]<\/p>\n \u201cIEDEREEN is zo geweldig geweest. Voor ons zijn jullie ALLEMAAL DOOR GOD GEZONDEN en we WAARDEREN alles wat jullie doen voor deze kleine engeltjes. Onze familie is zo overweldigd door opwinding en allerlei emoties met Adalia's reis naar Boston dit weekend, ik kan niet eens beginnen met het typen van de woorden van hoe we ons voelen.\u201d<\/em><\/p>\n \u201cDit nieuwe medicijn voor Zach geeft ons een hernieuwde hoop dat zijn hart sterker zal zijn, zijn glimlach stralender zal zijn en zijn leven langer zal duren. Deze nieuwe medicijnproef is een antwoord op onze gebeden. Dank aan iedereen die bij PRF betrokken is en dit mogelijk heeft gemaakt... de artsen, de onderzoekers en het personeel. Jullie zijn onze helden!\u201d<\/em><\/p>\n \u201cNamens Cam en onze familie, heel erg bedankt allemaal bij PRF voor alles wat jullie hebben gedaan! Zonder jullie waren we verdwaald in een wereld van verwarring en verdriet. In plaats daarvan leven we in een wereld van hoop en doel. Nogmaals bedankt! Met veel liefde en respect.\u201d\u00a0<\/em><\/p>\n [\/et_pb_testimonial][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_margin=”39px||” custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/trophies-3-kids.jpg” align=”center” align_tablet=”center” align_phone=”” align_last_edited=”on|desktop” admin_label=”aloha kids image” _builder_version=”4.22.1″ animation_style=”fade” animation_direction=”bottom” global_colors_info=”{}”] <\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|30px|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Summary, etc.” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”39px||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Onderzoekers identificeerden twee extra medicijnen die, wanneer ze worden gebruikt in combinatie met het huidige FTI-medicijn dat wordt getest (lonafarnib), een nog effectievere behandeling kunnen bieden voor kinderen met Progeria dan FTI's alleen. Pravastatine en zoledronaat werden toegevoegd aan de huidige behandeling lonafarnib. Deze veel grotere proef omvatte 45 kinderen uit 24 verschillende landen!<\/p>\n Alle drie de medicijnen richten zich op verschillende punten langs het pad dat leidt tot de productie van de ziekteverwekkende progerine. In opwindende laboratoriumstudies gepresenteerd door Dr. Carlos Lopez-Otin uit Spanje op de Progeria Research Foundation Scientific Workshop van 2007, verbeterden de twee nieuwe medicijnen de ziekte in Progeria-cellen en verlengden ze de levensduur in muismodellen van Progeria.<\/p>\n Als de drie in dit onderzoek toegediende medicijnen deze farnesylgroepbinding effectief kunnen blokkeren, dan kan progerine mogelijk worden "verlamd" en kan Progeria zelfs nog meer worden verbeterd dan met alleen lonafarnib. De hoop is dat de medicijnen als partners zouden werken, om elkaar aan te vullen, zodat het progerine-eiwit meer wordt be\u00efnvloed door de combinatie van de drie medicijnen.<\/p>\n Het team voerde een mini-onderzoek uit voor 5 kinderen met Progeria. Het korte, een maand durende 'haalbaarheidsonderzoek' vroeg of de combinatie van drie medicijnen goed verdragen zou worden, voordat er een groter internationaal onderzoek zou worden gestart. Bijwerkingen waren acceptabel en het team ging door naar het grotere effectiviteitsonderzoek.\u00a0\u00a0<\/strong><\/p>\n 45 kinderen namen deel aan deze proef, uit 24 landen, sprekend 17 talen. Dit omvat kinderen die deelnamen aan de FTI-only proef, de deelnemers aan de haalbaarheidsproef en andere kinderen die te jong waren om deel te nemen aan de eerste proef of kinderen die we ontdekten tijdens de eerste klinische proef (nadat de inschrijving was be\u00ebindigd). Kinderen die deelnamen aan de FTI-only proef hadden de mogelijkheid om deel te nemen aan de triple proef toen ze deelnamen aan hun laatste bezoek voor de huidige proef. Hierdoor konden die kinderen doorgaan met het nemen van FTI zonder dat ze een dosis misten.<\/p>\n [\/et_pb_text][et_pb_text admin_label=”Trial Medications at a Glance” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”39px||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Pravastatin<\/strong>\u00a0(op de markt gebracht als Pravachol of Selektine) is een lid van de klasse van statines. Het wordt meestal gebruikt om cholesterol te verlagen en hart- en vaatziekten te voorkomen.<\/p>\n Zoledroninezuur\u00a0<\/strong>is een\u00a0bisfosfonaat<\/strong>, meestal gebruikt als botmedicijn om osteoporose te verbeteren en om skeletbreuken te voorkomen bij mensen die lijden aan bepaalde vormen van kanker.<\/p>\n Lonafarnib <\/strong>is een\u00a0FTI\u00a0<\/strong>(Farnesyltransferase-remmer), een medicijn dat een afwijking in Progeria-cellen in het laboratorium kan terugdraaien en de ziekte bij Progeria-muizen heeft verbeterd. In juli een\u00a0studie\u00a0<\/a>** werd gepubliceerd waaruit bleek dat er geen significante verbeteringen werden gevonden ten opzichte van de enkelvoudige behandeling met lonafarnib. **Gordon, et. al., Klinische proef met prote\u00efnefarnesyleringsremmers lonafarnib, pravastatine en zoledroninezuur bij kinderen met Hutchinson-Gilford Progeria-syndroom, Circulatie<\/em>, 10.1161\/CIRCULATIEAHA.116.022188<\/strong><\/p>\n De \u201cTriple Trial\u201d werd echter verlengd na de oorspronkelijke periode van 2-3 jaar en uitgebreid tot maximaal 80 kinderen, zodat elk kind toegang zou kunnen hebben tot lonafarnib alleen, omdat we weten dat het de kinderen helpt. Normaal gesproken lopen klinische onderzoeken hun beloop en worden de pati\u00ebnten van alle medicijnen afgehaald totdat de FDA ze goedkeurt; dit kan jaren duren. PRF heeft ervoor gezorgd dat de kinderen de ene bekende behandeling blijven nemen, terwijl zij en hun onderzoekspartners aanvullende behandelingsopties blijven onderzoeken (zoals everolimus dat momenteel wordt getest).<\/p>\n [\/et_pb_text][et_pb_text admin_label=”Everolimus” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”||25px” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Everolimus is een vorm van het medicijn rapamycine; everolimus kan gemakkelijker worden gegeven aan kinderen met Progeria omdat er minder bloed hoeft te worden afgenomen om de medicijnspiegels te meten. Terwijl lonafarnib de ontwikkeling van het giftige progerine kan blokkeren, lijkt rapamycine cellen in staat te stellen progerine sneller op te ruimen. Dus met rapamycine gericht op een ander pad dan lonafarnib, kan de combinatie een "one-two punch" blijken te zijn voor Progeria - hopelijk een betere behandeling dan lonafarnib op zichzelf.<\/p>\n Rapamycine<\/em> is een door de FDA goedgekeurd medicijn waarvan eerder is aangetoond dat het de levensduur van niet-Progeria-muismodellen verlengt. Een studie* door onderzoekers van het NIH in Bethesda, MD en Massachusetts General Hospital in Boston toont aan dat rapamycine de hoeveelheid van het ziekteverwekkende eiwit progerine met 50% verlaagt, de abnormale kernvorm verbetert en de levensduur van Progeria-cellen in het laboratorium verlengt.<\/p>\n Rapamycine staat bekend om zijn anti-aging eigenschappen bij muizen. Deze bevindingen maken deel uit van een groeiende lijst van studies die helpen de theorie te valideren dat het vinden van de remedie voor Progeria ook de gehele ouder wordende bevolking ten goede kan komen.<\/p>\n \u00a0* K. Cao, J. J. Graziotto, C. D. Blair, J. R. Mazzulli, M. R. Erdos, D. Krainc, F. S. Collins, \u201cRapamycine keert cellulaire fenotypes om en verbetert de verwijdering van mutante eiwitten in Hutchinson-Gilford Progeria-syndroomcellen.\u201d Sci. Transl. Med. 3, 89ra58 (2011).<\/p>\n De Progeria Research Foundation heeft cellen voor dit project geleverd van de\u00a0PRF-cel- en weefselbank<\/a>\u00a0en hielp het onderzoek te financieren via onze\u00a0subsidieprogramma<\/a>\u00a0\u2013 nog meer bewijs dat de onderzoeksgerelateerde programma's van PRF essentieel zijn voor de vooruitgang op weg naar genezing.<\/p>\n Deze proef met 2 medicijnen was een gezamenlijke inspanning die voortbouwde op de kennis die was opgedaan bij eerdere klinische proeven van PRF. De kinderen werden gezien door vrijwel hetzelfde team van artsen van Boston Children's Hospital en Brigham and Women's Hospital, die nu allemaal wereldberoemde expertise hebben in Progeria en in de betrokken medicijnen.<\/p>\n Zestig kinderen uit 27 landen namen deel aan deze twee-medicijnfase. Gegevens van het twee-medicijngedeelte van de proef worden geanalyseerd en de resultaten worden geformuleerd en opgeschreven voor publicatie in een peer-reviewed wetenschappelijk tijdschrift.<\/p>\n [\/et_pb_text][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|30px|0px|false|false” custom_padding_tablet=”0px|35px|2px|35px|false|false” custom_padding_phone=”0px||0px” animation_direction=”right” border_width_top=”10px” border_color_top=”#8fd2ed” border_width_bottom=”10px” border_color_bottom=”#00b2e2″ global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Quest” _builder_version=”4.22.1″ header_4_line_height=”1.2em” custom_margin=”25px||25px” custom_padding=”||” animation_direction=”right” header_4_font_size_tablet=”” header_4_font_size_phone=”23px” header_4_font_size_last_edited=”on|phone” z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n Ons werk met genetische therapie\u00ebn gaat in volle gang! RNA-therapie en DNA-genbewerkingsstudies <\/strong>hebben een enorme Verbetering van de levensduur van Progeria-muizen. <\/strong>PRF blijft aanzienlijke fondsen investeren in hun ontwikkeling, in de hoop dat deze onderzoeksinspanningen zullen leiden tot leiden tot klinische proeven en uiteindelijk tot een geneesmiddel.<\/strong><\/p>\n Deze geavanceerde therapie\u00ebn hebben enorm potentieel<\/em>Met uw hulp kan PRF zo snel mogelijk doorgaan met het ontwikkelen van de meest effectieve behandelingen en genezing.<\/p>\n [\/et_pb_text][et_pb_button button_url=”https:\/\/www.progeriaresearch.org\/lonafarnib-map-program\/” button_text=”Patients, caregivers and doctors: Click here for details.” admin_label=”Lonafarnib pdf’s button” _builder_version=”4.22.1″ background_layout=”dark” custom_margin=”10px||10px||false|false” z_index_tablet=”500″ button_text_shadow_horizontal_length_tablet=”0px” button_text_shadow_vertical_length_tablet=”0px” button_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” box_shadow_spread_tablet=”0px” global_colors_info=”{}”] [\/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=\u201dDoneer nu\u201d admin_label=\u201dSamen vinden we de remedie!\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=\u201dop|bureaublad\u201d body_font_size_tablet=\u201d\u201d body_font_size_phone=\u201d\u201d body_font_size_last_edited=\u201dop|bureaublad\u201d z_index_tablet=\u201d500\u2033 border_radii=\u201dop|25px|25px|25px|25px\u201d global_colors_info=\u201d{}\u201d button_bg_color__hover_enabled=\u201daan\u201d button_bg_color__hover=\u201d#8fd2ed\u201d button_border_color__hover_enabled=\u201daan\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=\u201d2024 strip footer strip copy\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=”1″ fullwidth=”on” disabled_on=”off|off|off” _builder_version=”4.16″ border_width_bottom=”55px” border_color_bottom=”#29327a” locked=”off” global_colors_info=”{}”][et_pb_fullwidth_header _builder_version=”4.16″ title_font_size=”55px” background_color=”#29327a” background_image=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/09\/Kids-on-Bridge.png” custom_padding=”11.5vw||11.5vw||true” custom_padding_tablet=”” custom_padding_phone=”|56px||” 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;” title_text_shadow_horizontal_length_tablet=”0px” title_text_shadow_vertical_length_tablet=”0px” title_text_shadow_blur_strength_tablet=”1px” content_text_shadow_horizontal_length_tablet=”0px” content_text_shadow_vertical_length_tablet=”0px” content_text_shadow_blur_strength_tablet=”1px” subhead_text_shadow_horizontal_length_tablet=”0px” subhead_text_shadow_vertical_length_tablet=”0px” subhead_text_shadow_blur_strength_tablet=”1px” content_link_text_shadow_horizontal_length_tablet=”0px” content_link_text_shadow_vertical_length_tablet=”0px” content_link_text_shadow_blur_strength_tablet=”1px” content_ul_text_shadow_horizontal_length_tablet=”0px” content_ul_text_shadow_vertical_length_tablet=”0px” content_ul_text_shadow_blur_strength_tablet=”1px” content_ol_text_shadow_horizontal_length_tablet=”0px” content_ol_text_shadow_vertical_length_tablet=”0px” content_ol_text_shadow_blur_strength_tablet=”1px” content_quote_text_shadow_horizontal_length_tablet=”0px” content_quote_text_shadow_vertical_length_tablet=”0px” content_quote_text_shadow_blur_strength_tablet=”1px” button_one_text_shadow_horizontal_length_tablet=”0px” button_one_text_shadow_vertical_length_tablet=”0px” button_one_text_shadow_blur_strength_tablet=”1px” button_two_text_shadow_horizontal_length_tablet=”0px” button_two_text_shadow_vertical_length_tablet=”0px” button_two_text_shadow_blur_strength_tablet=”1px” box_shadow_horizontal_tablet=”0px” box_shadow_vertical_tablet=”0px” box_shadow_blur_tablet=”40px” […]<\/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\"]Start van een nieuw klinisch medicijnonderzoek: inschrijvingen voor progerinineonderzoek zijn begonnen!<\/em><\/strong><\/h4>\n
E-mailadres: sphillips@progeriaresearch.org<\/a>
WhatsApp, Telegram, WeChat: 1-978-876-2407
Kantoortelefoon: 978-548-5308<\/p>\nRNA-therapie: medicijntoediening<\/strong> Haalbaarheidsstudie is voltooid!<\/strong><\/h4>\n
Wat gebeurt er VANDAAG met de klinische proeven voor progeria?<\/strong><\/h2>\n
Procesgeschiedenis in \u00e9\u00e9n oogopslag<\/b><\/h2>\n
\n[\/et_pb_button][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”1_2,1_2″ custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_padding=”0|35px|0|0px|false|false” custom_padding_tablet=”0px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Details on the Prior Trials” _builder_version=”4.27.0″ global_colors_info=”{}”]<\/p>\nDetails over de eerdere rechtszaken<\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” custom_padding_last_edited=”on|desktop” padding_left_right_link_1=”true” padding_left_right_link_2=”true” _builder_version=”4.16″ custom_margin=”||25px” custom_padding=”|35px|0|0px|false|false” custom_padding_tablet=”35px|35px||35px” custom_padding_phone=”0px||0px” animation_direction=”right” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”May 7, 2007: The Start of the First-Ever Progeria Clinical Drug Trial Marks Historic Moment in Progeria Research History!” _builder_version=”4.27.2″ z_index_tablet=”500″ global_colors_info=”{}”]<\/p>\n7 mei 2007: De start van het allereerste klinische medicijnonderzoek naar progeria markeert een historisch moment in de geschiedenis van het progeriaonderzoek!<\/strong><\/h4>\n
\n[\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=”1_2″ saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|desktop” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”Twenty-eight (28) children from sixteen countries participated” _builder_version=”4.27.0″ custom_margin=”|||-30px|false|false” custom_margin_tablet=”35px||” custom_margin_phone=”” custom_margin_last_edited=”on|desktop” global_colors_info=”{}”]<\/p>\nWie, Waar, Wanneer, Hoe en Hoeveel\u2026<\/strong><\/h2>\n
Hoe zijn we op dit punt beland?<\/b><\/h4>\n
Waarom dachten onderzoekers dat dit medicijn zou werken bij Progeria?<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2019\/04\/FTI-3-cell-image-crpd-300x71.jpg\")
Hoe heeft PRF het onderzoek gefinancierd?
<\/strong><\/h4>\nDe FTI lonafarnib is nu een bewezen behandeling voor Progeria<\/strong>.<\/h2>\n
\n[\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row_inner column_structure=”undefined” use_custom_gutter=”on” gutter_width=”1″ module_id=”threeCT” _builder_version=”4.16″ background_color=”#29327a” custom_padding=”40px|0px|35.2344px|0px|false|false” animation_style=”slide” animation_direction=”right” animation_intensity_slide=”25%” border_width_bottom=”10px” border_color_bottom=”#8fd2ed” locked=”off” global_colors_info=”{}”][et_pb_column_inner type=”undefined” saved_specialty_column_type=”3_4″ _builder_version=”4.16″ custom_padding=”|||” custom_padding_tablet=”|0px||0px” custom_padding_phone=”||30px” custom_padding_last_edited=”on|phone” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_text admin_label=”The Progeria Triple Drug Trial Begins August 2009″ _builder_version=”4.22.1″ text_font=”||||||||” header_font=”||||||||” header_text_align=”center” header_text_color=”#ffffff” background_layout=”dark” animation_style=”fade” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” global_colors_info=”{}”]<\/p>\nAltijd vooruit: het Progeria Triple Drug Trial begint in augustus 2009<\/strong><\/h2>\n
Samenvatting:<\/strong><\/h4>\n
Strategie:<\/strong><\/h4>\n
Doel:<\/strong><\/h4>\n
Het haalbaarheidsonderzoek:<\/strong><\/h4>\n
Het effectiviteitsonderzoek:<\/strong><\/h4>\n
Onderzoeksmedicijnen in \u00e9\u00e9n oogopslag<\/strong><\/h4>\n
Alle drie de medicijnen blokkeren de productie van het farnesylmolecuul dat nodig is voor progerine om de ziekte Progeria te veroorzaken.
<\/em>
*<\/em>\u00a0\u201c<\/strong>Gecombineerde behandeling met statines en aminobisfosfonaten verlengt de levensduur in een muizenmodel van vroegtijdige veroudering bij de mens"<\/strong>, door 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 en Carlos Lopez-Ot\u00c4\u00b1n. Natuurgeneeskunde, 2008. 14(7): p. 767-72.<\/p>\nDe toevoeging van een nieuwere<\/strong> Medicijn: Everolimus<\/strong><\/h4>\n
De wetenschap achter de toevoeging van dit tweede medicijn<\/strong><\/h4>\n
![\"\"](\"https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2017\/05\/Cao-with-caption.jpg\")
<\/em><\/p>\nOnze zoektocht naar een remedie gaat door\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=”1″ module_class=”footer” _builder_version=”4.21.0″ background_color=”#29327a” custom_margin=”-2px|||||” custom_padding=”0|0px|0|0px|false|false” z_index_tablet=”500″ border_width_top=”12px” border_color_top=”#00b2e2″ global_module=”133″ locked=”off” global_colors_info=”{}”][et_pb_row_inner column_structure=”1_3,1_3,1_3″ disabled_on=”on|on|on” admin_label=”Row” _builder_version=”4.21.0″ _module_preset=”default” min_height=”277px” custom_margin=”|20px|30px||false|false” custom_padding=”0px|30px|0px||false|false” disabled=”on” global_colors_info=”{}”][et_pb_column_inner type=”1_3″ saved_specialty_column_type=”3_4″ _builder_version=”4.21.0″ _module_preset=”default” global_colors_info=”{}”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/08\/Startbanner-1.png” title_text=”Startbanner” url=”https:\/\/www.gravoc.com\/” align=”right” _builder_version=”4.21.0″ _module_preset=”default” custom_margin=”|-50px||20px|false|false” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column_inner][et_pb_column_inner type=”1_3″ saved_specialty_column_type=”3_4″ _builder_version=”4.21.0″ _module_preset=”default” global_colors_info=”{}”][\/et_pb_column_inner][et_pb_column_inner type=”1_3″ saved_specialty_column_type=”3_4″ _builder_version=”4.21.0″ _module_preset=”default” global_colors_info=”{}”][et_pb_image src=”https:\/\/www.progeriaresearch.org\/wp-content\/uploads\/2023\/07\/RFRFinish.png” title_text=”RFRFinish” _builder_version=”4.21.0″ _module_preset=”default” custom_margin=”|20px||-50px|false|false” global_colors_info=”{}”][\/et_pb_image][\/et_pb_column_inner][\/et_pb_row_inner][et_pb_row column_structure=”1_4,1_4,1_2″ make_equal=”on” module_class=” et_pb_row_fullwidth” _builder_version=”4.16″ width=”89%” width_tablet=”80%” width_phone=”” width_last_edited=”on|desktop” max_width=”89%” max_width_tablet=”80%” max_width_phone=”” max_width_last_edited=”on|desktop” z_index_tablet=”500″ make_fullwidth=”on” width_unit=”off” custom_width_percent=”100%” global_colors_info=”{}”][et_pb_column type=”1_4″ _builder_version=”4.16″ custom_padding=”|||” global_colors_info=”{}” custom_padding__hover=”|||”][et_pb_cta button_url=”https:\/\/www.progeriaresearch.org\/newsletter-signup\/” button_text=”Sign Up Now” admin_label=”Sign Up for Newsletter” module_class=”sign-btn” _builder_version=”4.16″ header_font_size=”25px” background_color=”#29327a” animation_style=”slide” animation_direction=”left” animation_intensity_slide=”25%” header_font_size_tablet=”” header_font_size_phone=”30px” header_font_size_last_edited=”on|desktop” z_index_tablet=”500″ 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>\nAanmelden<\/h2>\n
voor onze<\/h2>\n
Nieuwsbrief!<\/h2>\n
Samen, wij<\/h2>\n
ZULLEN<\/em><\/h2>\n
vind de remedie!<\/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