{"id":2612,"date":"2025-01-27T11:49:18","date_gmt":"2025-01-27T08:49:18","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2612"},"modified":"2025-01-27T14:06:33","modified_gmt":"2025-01-27T11:06:33","slug":"roche-announces-new-results-from-the-embark-study-of-elevidys-in-outpatients-with-duchenne-muscular-dystrophy-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/roche-announces-new-results-from-the-embark-study-of-elevidys-in-outpatients-with-duchenne-muscular-dystrophy-dmd\/","title":{"rendered":"Roche annonce de nouveaux r\u00e9sultats de l&#039;\u00e9tude EMBARK sur le g\u00e8ne Elevidys chez des patients ambulatoires atteints de dystrophie musculaire de Duchenne (DMD)"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Roche a d\u00e9voil\u00e9 aujourd&#039;hui des donn\u00e9es encourageantes issues de la deuxi\u00e8me ann\u00e9e de l&#039;essai EMBARK, une \u00e9tude mondiale de phase III randomis\u00e9e en double aveugle sur Elevidys (delandistrogene moxeparvovec), la premi\u00e8re th\u00e9rapie g\u00e9nique autoris\u00e9e pour le traitement de la dystrophie musculaire de Duchenne.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Compar\u00e9s \u00e0 un groupe t\u00e9moin externe non trait\u00e9 et pond\u00e9r\u00e9 par la propension pr\u00e9d\u00e9termin\u00e9e, des am\u00e9liorations statistiquement et cliniquement significatives ont \u00e9t\u00e9 observ\u00e9es dans trois tests de fonction motrice importants deux ans apr\u00e8s le traitement par Elevidys : NSAA, TTR et 10MWR. Entre un et deux ans apr\u00e8s l&#039;administration de la dose, les diff\u00e9rences fonctionnelles entre les personnes recevant Elevidys et celles du groupe t\u00e9moin externe se sont accrues. Dans leur ensemble, ces r\u00e9sultats montrent que Elevidys a syst\u00e9matiquement b\u00e9n\u00e9fici\u00e9.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u00ab Apr\u00e8s deux ans de traitement avec Elevidys, nous constatons de nombreux b\u00e9n\u00e9fices durables dans la vie quotidienne de ces jeunes gar\u00e7ons, qui sont tous des indicateurs du potentiel de modification de la maladie dans la maladie de Duchenne \u00bb, a d\u00e9clar\u00e9 Levi Garraway, MD, Ph.D., directeur m\u00e9dical et responsable du d\u00e9veloppement mondial des produits de Roche. \u00ab Ces r\u00e9sultats, qui incluent des am\u00e9liorations dans la station debout, la marche et la course, repr\u00e9sentent des progr\u00e8s significatifs et nous pr\u00e9voyons de les partager avec les autorit\u00e9s sanitaires le plus rapidement possible. \u00bb<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>En savoir plus:<\/strong> <a href=\"https:\/\/dmdwarrior.com\/fr\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Questions fr\u00e9quemment pos\u00e9es sur Elevidys<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Today, Roche revealed encouraging topline data from the second year of the EMBARK trial, a worldwide, double-blind, randomised phase III investigation of Elevidys (delandistrogene moxeparvovec), the first gene therapy to be authorized for the treatment of Duchenne muscular dystrophy. When compared to a predetermined propensity-weighted untreated external control group, statistically significant and clinically meaningful improvements [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2613,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[66,33,207,61,203,65],"class_list":["post-2612","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-duchenne-muscular-dystrophy","tag-elevidys","tag-embark","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2612","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=2612"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/2612\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/2613"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=2612"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=2612"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=2612"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}