{"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\/es\/elevidys-gene-therapy-for-non-ambulant-children-with-dmd-fda-oks-endeavor-cohort-8-immunosuppression-study\/","title":{"rendered":"Terapia g\u00e9nica ELEVIDYS para ni\u00f1os con DMD que no pueden caminar: FDA aprueba el estudio de inmunosupresi\u00f3n de la cohorte 8 de ENDEAVOR"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Sarepta Therapeutics confirm\u00f3 que FDA ha autorizado el inicio de la dosificaci\u00f3n en la cohorte 8 del ensayo ENDEAVOR (estudio 9001-103).<\/strong> Esta cohorte crucial est\u00e1 dise\u00f1ada espec\u00edficamente para evaluar un r\u00e9gimen inmunosupresor mejorado junto con el tratamiento con ELEVIDYS (delandistr\u00f3geno moxeparvovec-rokl) para pacientes con distrofia muscular de Duchenne (DMD) no ambulantes.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ensayo ENDEAVOR (Estudio 9001-103)<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Aproximadamente 25 participantes no ambulatorios recibir\u00e1n sirolimus como parte del r\u00e9gimen en la cohorte 8 del estudio ENDEAVOR, que se espera que comience antes de fin de a\u00f1o.<\/li>\n\n\n\n<li>El r\u00e9gimen inmunosupresor mejorado est\u00e1 dise\u00f1ado para mitigar el riesgo de lesi\u00f3n hep\u00e1tica aguda (ALI) e insuficiencia hep\u00e1tica aguda (ALF) asociadas con la terapia g\u00e9nica AAV.<\/li>\n\n\n\n<li>Las decisiones sobre la reanudaci\u00f3n de la dosificaci\u00f3n comercial para esta poblaci\u00f3n se tomar\u00e1n en colaboraci\u00f3n con FDA despu\u00e9s de revisar los datos del estudio.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Los datos de la cohorte 8 se utilizar\u00e1n para determinar si la administraci\u00f3n de sirolimus antes y despu\u00e9s de la infusi\u00f3n de ELEVIDYS puede ayudar a reducir la lesi\u00f3n hep\u00e1tica aguda (ALI), un riesgo conocido asociado con la terapia g\u00e9nica con AAV.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">La cohorte incluir\u00e1 a aproximadamente 25 participantes no ambulatorios en EE. UU. El r\u00e9gimen de inmunosupresi\u00f3n incluir\u00e1 14 d\u00edas de administraci\u00f3n periinfusi\u00f3n de sirolimus (antes de la administraci\u00f3n de ELEVIDYS) y continuar\u00e1 durante 12 semanas despu\u00e9s de la administraci\u00f3n de ELEVIDYS.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>M\u00e1s informaci\u00f3n<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/es\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">Preguntas frecuentes sobre 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"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/5229","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/comments?post=5229"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/posts\/5229\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media\/5231"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/media?parent=5229"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/categories?post=5229"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/es\/wp-json\/wp\/v2\/tags?post=5229"}],"curies":[{"name":"gracias","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}