{"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\/pt\/elevidys-gene-therapy-for-non-ambulant-children-with-dmd-fda-oks-endeavor-cohort-8-immunosuppression-study\/","title":{"rendered":"Terapia g\u00eanica ELEVIDYS para crian\u00e7as n\u00e3o ambulantes com DMD: FDA aprova estudo de imunossupress\u00e3o da coorte 8 com ENDEAVOR"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>O estudo Sarepta Therapeutics confirmou que o estudo FDA autorizou o in\u00edcio da administra\u00e7\u00e3o da dose na Coorte 8 do ensaio cl\u00ednico ENDEAVOR (Estudo 9001-103).<\/strong> Esta coorte crucial foi especificamente concebida para avaliar um regime imunossupressor refor\u00e7ado em conjunto com o tratamento com ELEVIDYS (delandistrogene moxeparvovec-rokl) para pacientes n\u00e3o ambulantes com distrofia muscular de Duchenne (DMD).<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Ensaio ENDEAVOR (Estudo 9001-103)<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Aproximadamente 25 participantes n\u00e3o ambulatoriais receber\u00e3o sirolimus como parte do regime terap\u00eautico na Coorte 8 do estudo ENDEAVOR, que dever\u00e1 come\u00e7ar antes do final do ano.<\/li>\n\n\n\n<li>O regime imunossupressor refor\u00e7ado visa mitigar o risco de les\u00e3o hep\u00e1tica aguda (LHA) e insufici\u00eancia hep\u00e1tica aguda (IHA) associadas \u00e0 terapia g\u00eanica com AAV.<\/li>\n\n\n\n<li>As decis\u00f5es relativas \u00e0 retomada da administra\u00e7\u00e3o comercial da dose para essa popula\u00e7\u00e3o ser\u00e3o tomadas em colabora\u00e7\u00e3o com a FDA ap\u00f3s a revis\u00e3o dos dados do estudo.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">Os dados da Coorte 8 ser\u00e3o usados para determinar se a administra\u00e7\u00e3o de sirolimus antes e depois da infus\u00e3o de ELEVIDYS pode ajudar a reduzir a les\u00e3o hep\u00e1tica aguda (LHA), um risco conhecido associado \u00e0 terapia g\u00eanica com AAV.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">O estudo de coorte incluir\u00e1 aproximadamente 25 participantes nos EUA que n\u00e3o sejam ambulatoriais. O regime de imunossupress\u00e3o incluir\u00e1 14 dias de administra\u00e7\u00e3o de sirolimus no per\u00edodo peri-infus\u00e3o (antes da administra\u00e7\u00e3o de ELEVIDYS) e continuar\u00e1 por 12 semanas ap\u00f3s a administra\u00e7\u00e3o de ELEVIDYS.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Saber mais<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/pt\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">Perguntas frequentes 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\/pt\/wp-json\/wp\/v2\/posts\/5229","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=5229"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/5229\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/5231"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=5229"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=5229"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=5229"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}