{"id":4237,"date":"2025-07-29T09:36:33","date_gmt":"2025-07-29T06:36:33","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4237"},"modified":"2025-07-29T09:36:34","modified_gmt":"2025-07-29T06:36:34","slug":"fda-elevidys-gene-therapy-will-continue-to-be-used-in-ambulatory-dmd-patients","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/fda-elevidys-gene-therapy-will-continue-to-be-used-in-ambulatory-dmd-patients\/","title":{"rendered":"FDA\uff1a\u201cElevidys\u57fa\u56e0\u7597\u6cd5\u5c06\u7ee7\u7eed\u7528\u4e8e\u95e8\u8bcaDMD\u60a3\u8005\u201d"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Elevidys \u662f\u4e00\u79cd\u7531 Sarepta Therapeutics \u57fa\u56e0\u7597\u6cd5\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD)\uff0c\u73b0\u5df2\u53ef\u4f9b\u95e8\u8bca\u60a3\u8005\u4f7f\u7528\uff0c\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u5efa\u8bae\u89e3\u9664\u81ea\u613f\u6682\u505c\u4e0a\u5e02\u7684\u653f\u7b56\u3002FDA \u57fa\u56e0\u7597\u6cd5\u7684\u8c03\u67e5\u53d1\u73b0\uff0c\u8be5\u57fa\u56e0\u7597\u6cd5\u4ea7\u54c1\u4e0e\u8fd9\u540d 8 \u5c81\u7537\u5b69\u7684\u6b7b\u4ea1\u65e0\u5173\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sarepta \u7684\u57fa\u56e0\u7597\u6cd5\u9879\u76ee\u59cb\u4e8e\u4eca\u5e74\u65e9\u4e9b\u65f6\u5019\uff0c\u5f53\u65f6\u51fa\u73b0\u4e86\u4e25\u91cd\u7684\u5b89\u5168\u9690\u60a3\u548c\u6b7b\u4ea1\u75c5\u4f8b\u3002\u5982\u4eca\uff0c\u7531\u4e8e\u76d1\u7ba1\u90e8\u95e8\u7684\u4e25\u5389\u6253\u51fb\uff0c\u8be5\u9879\u76ee\u53d1\u751f\u4e86\u65b0\u7684\u8f6c\u6298\u3002\u5728\u7b2c\u4e09\u540d\u60a3\u8005\uff08\u4e00\u540d 51 \u5c81\u7537\u6027\uff0c\u53c2\u4e0e\u4e86\u8be5\u836f\u7269\u6cbb\u7597\u80a2\u5e26\u578b\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 I \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u8be5\u8bd5\u9a8c\u4e5f\u4f7f\u7528\u4e86 AAV \u8f7d\u4f53\uff09\u6b7b\u4ea1\u540e\uff0cFDA \u516c\u53f8\u4ecb\u5165\u5e76\u8981\u6c42 Sarepta \u6682\u505c\u5728\u7f8e\u56fd\u5411\u6240\u6709\u95e8\u8bca\u60a3\u8005\u63d0\u4f9b\u8be5\u836f\u7269\u3002\u5468\u4e94\u53c8\u62a5\u544a\u4e86\u4e00\u8d77\u6b7b\u4ea1\u75c5\u4f8b\uff0cFDA \u516c\u53f8\u968f\u5373\u542f\u52a8\u4e86\u8c03\u67e5\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0cFDA \u57fa\u56e0\u7597\u6cd5\u516c\u53f8\u5728\u5468\u4e00\u4e0b\u5348\u53d1\u5e03\u7684\u4e00\u4efd\u7b80\u660e\u58f0\u660e\u4e2d\u5efa\u8bae\u89e3\u9664\u81ea\u613f\u6682\u505c\uff0c\u5141\u8bb8\u95e8\u8bca\u60a3\u8005\u7ee7\u7eed\u670d\u7528 Elevidys \u57fa\u56e0\u7597\u6cd5\u3002\u8428\u96f7\u666e\u5854\u516c\u53f8\u548c\u5df4\u897f\u5f53\u5c40\u4e00\u81f4\u8ba4\u4e3a\uff0c\u8fd9\u540d\u516b\u5c81\u5df4\u897f\u60a3\u8005\u6700\u8fd1\u7684\u6b7b\u4ea1\u5f88\u53ef\u80fd\u662f\u7531\u4e25\u91cd\u7684\u6d41\u611f\u53d1\u4f5c\u5f15\u8d77\u7684\uff0c\u800c\u975e\u57fa\u56e0\u7597\u6cd5\u4ea7\u54c1\u672c\u8eab\u3002\u8fd9\u79cd\u611f\u67d3\u4f1a\u56e0\u514d\u75ab\u6291\u5236\u800c\u6076\u5316\u3002\u2014\u2014\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/fda-investigating-death-of-8-year-old-brazilian-boy-who-received-elevidys\/\">FDA \u8c03\u67e5\u63a5\u53d7 Elevidys \u75ab\u82d7\u7684 8 \u5c81\u5df4\u897f\u7537\u5b69\u7684\u6b7b\u4ea1<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bf9\u4e8e\u65e0\u6cd5\u884c\u8d70\u7684\u60a3\u8005\uff0c\u81ea\u613f\u6682\u505c\u6cbb\u7597\u4ecd\u7136\u6709\u6548\uff0c\u5728 Elevidys \u53d1\u751f\u4e24\u8d77\u6b7b\u4ea1\u4e8b\u4ef6\u540e\uff0cFDA \u5c1a\u672a\u9488\u5bf9\u8fd9\u4e9b\u60a3\u8005\u53d1\u5e03\u4efb\u4f55\u5efa\u8bae\u3002<\/p>","protected":false},"excerpt":{"rendered":"<p>Elevidys, a gene therapy for Duchenne Muscular Dystrophy (DMD) from Sarepta Therapeutics, is now available to ambulatory patients, and the U.S. Food and Drug Administration is suggesting that the voluntary hold be lifted. The FDA&#8217;s inquiry found that the gene therapy product had nothing to do with the 8-year-old boy&#8217;s death. The story of Sarepta&#8217;s [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4239,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[33,179,88,61,203,65],"class_list":["post-4237","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-elevidys","tag-elevidys-gene-therapy","tag-fda","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4237","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=4237"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4237\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4239"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4237"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4237"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4237"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}