{"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\/tr\/elevidys-gene-therapy-for-non-ambulant-children-with-dmd-fda-oks-endeavor-cohort-8-immunosuppression-study\/","title":{"rendered":"DMD&#039;li Y\u00fcr\u00fcyemeyen \u00c7ocuklar i\u00e7in ELEVIDYS Gen Terapisi: FDA, ENDEAVOR Kohort 8 \u0130mm\u00fcnosupresyon \u00c7al\u0131\u015fmas\u0131n\u0131 Onaylad\u0131"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Sarepta Therapeutics, FDA&#039;nin ENDEAVOR (\u00c7al\u0131\u015fma 9001-103) \u00e7al\u0131\u015fmas\u0131n\u0131n 8. Kohortunda dozlaman\u0131n ba\u015flamas\u0131n\u0131 onaylad\u0131\u011f\u0131n\u0131 do\u011frulad\u0131.<\/strong> Bu kritik kohort, hareket edemeyen Duchenne kas distrofisi (DMD) hastalar\u0131nda ELEVIDYS (delandistrogene moxeparvovec-rokl) tedavisinin yan\u0131 s\u0131ra g\u00fc\u00e7lendirilmi\u015f bir imm\u00fcnos\u00fcpresif rejimin de\u011ferlendirilmesi i\u00e7in \u00f6zel olarak tasarlanm\u0131\u015ft\u0131r.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">ENDEAVOR (\u00c7al\u0131\u015fma 9001-103) Denemesi<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>Y\u0131l sonundan \u00f6nce ba\u015flamas\u0131 beklenen ENDEAVOR \u00e7al\u0131\u015fmas\u0131n\u0131n 8. Kohortundaki rejimin bir par\u00e7as\u0131 olarak yakla\u015f\u0131k 25 y\u00fcr\u00fcyemeyen kat\u0131l\u0131mc\u0131ya sirolimus verilecektir.<\/li>\n\n\n\n<li>Geli\u015ftirilmi\u015f imm\u00fcnos\u00fcpresif rejim, AAV gen tedavisiyle ili\u015fkili akut karaci\u011fer hasar\u0131 (ALI) ve akut karaci\u011fer yetmezli\u011fi (ALF) riskini azaltmak i\u00e7in tasarlanm\u0131\u015ft\u0131r<\/li>\n\n\n\n<li>Bu pop\u00fclasyon i\u00e7in ticari dozaj\u0131n yeniden ba\u015flat\u0131lmas\u0131na ili\u015fkin kararlar, \u00e7al\u0131\u015fma verilerinin incelenmesinden sonra FDA ile i\u015f birli\u011fi i\u00e7inde al\u0131nacakt\u0131r.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">8. Kohorttan elde edilen veriler, sirolimusun ELEVIDYS inf\u00fczyonundan \u00f6nce ve sonra uygulanmas\u0131n\u0131n, AAV gen tedavisiyle ili\u015fkili bilinen bir risk olan akut karaci\u011fer hasar\u0131n\u0131 (ALI) azaltmaya yard\u0131mc\u0131 olup olamayaca\u011f\u0131n\u0131 belirlemek i\u00e7in kullan\u0131lacakt\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Kohorta, ABD&#039;de hareket kabiliyeti olmayan yakla\u015f\u0131k 25 kat\u0131l\u0131mc\u0131 dahil edilecektir. \u0130mm\u00fcns\u00fcpresyon rejimi, ELEVIDYS uygulamas\u0131ndan \u00f6nce 14 g\u00fcn peri-inf\u00fczyon sirolimus dozlamas\u0131n\u0131 i\u00e7erecek ve ELEVIDYS uygulamas\u0131ndan sonra 12 hafta boyunca devam edecektir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Daha fazla bilgi edin<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/tr\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">Elevidys Hakk\u0131nda S\u0131k\u00e7a Sorulan Sorular<\/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\/tr\/wp-json\/wp\/v2\/posts\/5229","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=5229"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/5229\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/5231"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=5229"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=5229"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=5229"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}