{"id":5484,"date":"2026-01-26T16:38:21","date_gmt":"2026-01-26T13:38:21","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5484"},"modified":"2026-01-26T23:15:59","modified_gmt":"2026-01-26T20:15:59","slug":"sarepta-3-year-topline-data-of-elevidys-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/sarepta-3-year-topline-data-of-elevidys-gene-therapy\/","title":{"rendered":"Sarepta a publi\u00e9 les principaux r\u00e9sultats \u00e0 3 ans de l'\u00e9tude ELEVIDYS sur la th\u00e9rapie g\u00e9nique chez des patients ambulatoires atteints de dystrophie musculaire de Duchenne"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Sarepta a annonc\u00e9 les principaux r\u00e9sultats fonctionnels \u00e0 3 ans des patients trait\u00e9s dans la premi\u00e8re partie d&#039;EMBARK (\u00e9tude 9001-301), l&#039;\u00e9tude mondiale de phase 3 randomis\u00e9e contr\u00f4l\u00e9e par placebo \u00e9valuant ELEVIDYS (delandistrogene moxeparvovec-rokl) chez des personnes ambulatoires atteintes de dystrophie musculaire de Duchenne \u00e2g\u00e9es de quatre \u00e0 sept ans au moment du traitement.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Les niveaux de cr\u00e9atine kinase, le biomarqueur le plus important de la dystrophie musculaire de Duchenne, n&#039;ont pas \u00e9t\u00e9 divulgu\u00e9s par Sarepta dans les donn\u00e9es sur 3 ans.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u00c0 un \u00e2ge moyen de 9 ans, les patients trait\u00e9s par ELEVIDYS ont atteint des scores moyens d&#039;\u00e9valuation ambulatoire North Star (NSAA) sup\u00e9rieurs \u00e0 la valeur de r\u00e9f\u00e9rence trois ans apr\u00e8s le traitement (n=52).<\/li>\n\n\n\n<li>La th\u00e9rapie g\u00e9nique ELEVIDYS a d\u00e9montr\u00e9 une r\u00e9duction de 70% ou plus du taux de d\u00e9clin par rapport au groupe t\u00e9moin externe pond\u00e9r\u00e9 par la propension, mesur\u00e9e par le \u201c\u00a0temps de lever\u00a0\u201d (TTR) et le test de marche\/course de 10 m\u00e8tres (10MWR). Les patients trait\u00e9s par ELEVIDYS ont pr\u00e9sent\u00e9 un effet th\u00e9rapeutique croissant au fil du temps, l\u2019\u00e9cart fonctionnel par rapport au groupe t\u00e9moin externe s\u2019\u00e9largissant significativement entre la 2e et la 3e ann\u00e9e.<\/li>\n\n\n\n<li>Aucun nouveau signal de s\u00e9curit\u00e9 li\u00e9 au traitement n&#039;a \u00e9t\u00e9 observ\u00e9, ce qui est coh\u00e9rent avec le profil de s\u00e9curit\u00e9 acceptable observ\u00e9 jusqu&#039;\u00e0 pr\u00e9sent avec ELEVIDYS chez les patients ambulatoires.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/fr\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u00c9valuation ambulatoire North Star (NSAA) dans la dystrophie musculaire de Duchenne<\/a><\/p>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"778\" height=\"118\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png\" alt=\"Sarepta a publi\u00e9 les principaux r\u00e9sultats \u00e0 3 ans de l&#039;\u00e9tude ELEVIDYS sur la th\u00e9rapie g\u00e9nique chez des patients ambulatoires atteints de dystrophie musculaire de Duchenne\" class=\"wp-image-5496\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png 778w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-300x46.png 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-768x116.png 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-18x3.png 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-150x23.png 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-696x106.png 696w\" sizes=\"auto, (max-width: 778px) 100vw, 778px\" \/><\/figure>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td><strong>R\u00e9sultats fonctionnels<\/strong><\/td><td><strong>Diff\u00e9rence de changement LSM vs EC<\/strong><\/td><td><strong>Valeur p<\/strong><\/td><\/tr><tr><td>NSAA<\/td><td>+4,39 points (am\u00e9lioration)<\/td><td>p=0,0002<\/td><\/tr><tr><td>TTR<\/td><td>-6,05 secondes (am\u00e9lioration)<\/td><td>p&lt;0,0001<\/td><\/tr><tr><td>10MWR<\/td><td>-2,70 secondes (am\u00e9lioration)<\/td><td>p=0,0039<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">Le 26 janvier 2026, l&#039;action Sarepta a d\u00e9but\u00e9 \u00e0 22 dollars am\u00e9ricains, puis a grimp\u00e9 \u00e0 24,68 dollars am\u00e9ricains suite \u00e0 la publication des donn\u00e9es sur trois ans de son traitement de th\u00e9rapie g\u00e9nique Elevidys. Cependant, quelques heures plus tard, elle a chut\u00e9 \u00e0 22,52 dollars am\u00e9ricains \u00e0 13h04, heure de l&#039;Est. Ce repli a \u00e9t\u00e9 interpr\u00e9t\u00e9 comme un signe d&#039;insatisfaction des investisseurs quant aux donn\u00e9es triennales relatives \u00e0 la th\u00e9rapie g\u00e9nique Elevidys.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Apprendre encore plus:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/fr\/creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Taux de cr\u00e9atine kinase (CK), de dystrophine et score NSAA dans la th\u00e9rapie g\u00e9nique Elevidys<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Sarepta announced 3-year topline functional results from patients treated in Part One of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment. Sarepta were not disclosed creatine kinase levels, the most major biomarker [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5485,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[66,33,179,65],"class_list":["post-5484","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duchenne-muscular-dystrophy","tag-elevidys","tag-elevidys-gene-therapy","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5484","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/comments?post=5484"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5484\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/5485"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=5484"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=5484"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=5484"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}