{"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\/zh\/elevidys-gene-therapy-for-non-ambulant-children-with-dmd-fda-oks-endeavor-cohort-8-immunosuppression-study\/","title":{"rendered":"ELEVIDYS\u57fa\u56e0\u7597\u6cd5\u6cbb\u7597\u975e\u884c\u8d70\u578bDMD\u60a3\u513f\uff1aFDA OKs ENDEAVOR \u7b2c8\u7ec4\u514d\u75ab\u6291\u5236\u7814\u7a76"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Sarepta Therapeutics \u786e\u8ba4 FDA \u5df2\u6279\u51c6 ENDEAVOR\uff089001-103 \u7814\u7a76\uff09\u8bd5\u9a8c\u7b2c 8 \u7ec4\u5f00\u59cb\u7ed9\u836f\u3002.<\/strong> \u8be5\u5173\u952e\u961f\u5217\u4e13\u95e8\u7528\u4e8e\u8bc4\u4f30\u589e\u5f3a\u514d\u75ab\u6291\u5236\u65b9\u6848\u4e0e ELEVIDYS\uff08delandistrogene moxeparvovec-rokl\uff09\u6cbb\u7597\u5bf9\u975e\u884c\u8d70\u578b\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u7684\u7597\u6548\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">ENDEAVOR\uff08\u7814\u7a76 9001-103\uff09\u8bd5\u9a8c<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5927\u7ea625\u540d\u65e0\u6cd5\u884c\u8d70\u7684\u53c2\u4e0e\u8005\u5c06\u5728ENDEAVOR\u7814\u7a76\u7684\u7b2c8\u7ec4\u961f\u5217\u4e2d\u63a5\u53d7\u897f\u7f57\u83ab\u53f8\u6cbb\u7597\uff0c\u8be5\u961f\u5217\u9884\u8ba1\u5c06\u4e8e\u5e74\u5e95\u524d\u5f00\u59cb\u3002<\/li>\n\n\n\n<li>\u52a0\u5f3a\u514d\u75ab\u6291\u5236\u65b9\u6848\u65e8\u5728\u964d\u4f4eAAV\u57fa\u56e0\u6cbb\u7597\u76f8\u5173\u7684\u6025\u6027\u809d\u635f\u4f24\uff08ALI\uff09\u548c\u6025\u6027\u809d\u8870\u7aed\uff08ALF\uff09\u98ce\u9669\u3002<\/li>\n\n\n\n<li>\u5173\u4e8e\u662f\u5426\u6062\u590d\u5bf9\u8be5\u4eba\u7fa4\u8fdb\u884c\u5546\u4e1a\u7ed9\u836f\u7684\u51b3\u5b9a\uff0c\u5c06\u5728\u5ba1\u67e5\u7814\u7a76\u6570\u636e\u540e\u4e0e FDA \u5408\u4f5c\u505a\u51fa\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u7b2c 8 \u7ec4\u7684\u6570\u636e\u5c06\u7528\u4e8e\u786e\u5b9a\u5728 ELEVIDYS \u8f93\u6ce8\u4e4b\u524d\u548c\u4e4b\u540e\u7ed9\u4e88\u897f\u7f57\u83ab\u53f8\u662f\u5426\u53ef\u4ee5\u5e2e\u52a9\u51cf\u5c11\u6025\u6027\u809d\u635f\u4f24 (ALI)\uff0c\u8fd9\u662f\u4e0e AAV \u57fa\u56e0\u6cbb\u7597\u76f8\u5173\u7684\u5df2\u77e5\u98ce\u9669\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u961f\u5217\u7814\u7a76\u5c06\u5728\u7f8e\u56fd\u62db\u52df\u7ea6 25 \u540d\u65e0\u6cd5\u884c\u8d70\u7684\u53c2\u4e0e\u8005\u3002\u514d\u75ab\u6291\u5236\u65b9\u6848\u5305\u62ec\u8f93\u6ce8\u897f\u7f57\u83ab\u53f8 14 \u5929\uff08\u5728 ELEVIDYS \u7ed9\u836f\u524d\uff09\uff0c\u5e76\u5728 ELEVIDYS \u7ed9\u836f\u540e\u7ee7\u7eed\u670d\u7528 12 \u5468\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\">\u5173\u4e8e Elevidys \u7684\u5e38\u89c1\u95ee\u9898\u89e3\u7b54<\/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\/zh\/wp-json\/wp\/v2\/posts\/5229","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/comments?post=5229"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5229\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5231"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5229"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5229"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5229"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}