{"id":5229,"date":"2025-11-25T17:51:34","date_gmt":"2025-11-25T14:51:34","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5229"},"modified":"2025-11-25T18:10:05","modified_gmt":"2025-11-25T15:10:05","slug":"elevidys-gene-therapy-for-non-ambulant-children-with-dmd-fda-oks-endeavor-cohort-8-immunosuppression-study","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/fr\/elevidys-gene-therapy-for-non-ambulant-children-with-dmd-fda-oks-endeavor-cohort-8-immunosuppression-study\/","title":{"rendered":"\u00c9tude de th\u00e9rapie g\u00e9nique ELEVIDYS pour les enfants non ambulatoires atteints de DMD\u00a0: FDA est compatible avec ENDEAVOR. \u00c9tude d\u2019immunosuppression de la cohorte 8."},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Sarepta Therapeutics a confirm\u00e9 que FDA a autoris\u00e9 le d\u00e9but de l&#039;administration du dosage dans la cohorte 8 de l&#039;essai ENDEAVOR (\u00e9tude 9001-103).<\/strong> Cette cohorte cruciale est sp\u00e9cifiquement con\u00e7ue pour \u00e9valuer un r\u00e9gime immunosuppresseur am\u00e9lior\u00e9 parall\u00e8lement au traitement ELEVIDYS (d\u00e9landistrog\u00e8ne mox\u00e9parvovec-rokl) pour les patients atteints de dystrophie musculaire de Duchenne (DMD) non ambulatoires.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Essai ENDEAVOR (\u00c9tude 9001-103)<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Environ 25 participants non ambulatoires recevront du sirolimus dans le cadre du traitement de la cohorte 8 de l&#039;\u00e9tude ENDEAVOR, dont le d\u00e9but est pr\u00e9vu avant la fin de l&#039;ann\u00e9e.<\/li>\n\n\n\n<li>Le protocole immunosuppresseur renforc\u00e9 est con\u00e7u pour att\u00e9nuer le risque de l\u00e9sion h\u00e9patique aigu\u00eb (LHA) et d&#039;insuffisance h\u00e9patique aigu\u00eb (IHA) associ\u00e9es \u00e0 la th\u00e9rapie g\u00e9nique par AAV.<\/li>\n\n\n\n<li>Les d\u00e9cisions concernant la reprise de l&#039;administration commerciale du traitement \u00e0 cette population seront prises en collaboration avec FDA apr\u00e8s examen des donn\u00e9es de l&#039;\u00e9tude.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Les donn\u00e9es de la cohorte 8 seront utilis\u00e9es pour d\u00e9terminer si l&#039;administration de sirolimus avant et apr\u00e8s la perfusion d&#039;ELEVIDYS peut aider \u00e0 r\u00e9duire les l\u00e9sions h\u00e9patiques aigu\u00ebs (LHA), un risque connu associ\u00e9 \u00e0 la th\u00e9rapie g\u00e9nique AAV.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">La cohorte inclura environ 25 participants non ambulatoires aux \u00c9tats-Unis. Le traitement immunosuppresseur comprendra 14 jours d&#039;administration de sirolimus en p\u00e9ri-perfusion (avant l&#039;administration d&#039;ELEVIDYS) et se poursuivra pendant 12 semaines apr\u00e8s l&#039;administration d&#039;ELEVIDYS.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Apprendre encore plus<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/fr\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">Questions fr\u00e9quemment pos\u00e9es sur Elevidys<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Sarepta Therapeutics confirmed that the FDA has sanctioned the start of dosing in Cohort 8 of the ENDEAVOR (Study 9001-103) trial. This crucial cohort is specifically designed to assess an enhanced immunosuppressive regimen alongside ELEVIDYS (delandistrogene moxeparvovec-rokl) treatment for non-ambulant Duchenne muscular dystrophy (DMD) patients. ENDEAVOR (Study 9001-103) Trial Data from Cohort 8 will be [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5231,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[121,33,179,61,65],"class_list":["post-5229","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-elevidys-gene-therapy","tag-gene-therapy","tag-sarepta"],"subtitle":"Sarepta a annonc\u00e9 que le FDA a approuv\u00e9 l'\u00e9tude ENDEAVOR (9001-103) pour son lancement.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5229","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=5229"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/posts\/5229\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media\/5231"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/media?parent=5229"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/categories?post=5229"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/fr\/wp-json\/wp\/v2\/tags?post=5229"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}