{"id":3925,"date":"2025-06-15T12:00:44","date_gmt":"2025-06-15T09:00:44","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3925"},"modified":"2025-06-15T12:00:45","modified_gmt":"2025-06-15T09:00:45","slug":"sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/sarepta-reported-elevidys-gene-therapy-for-duchenne-muscular-dystrophy-will-not-be-used-in-non-ambulatory-patients\/","title":{"rendered":"Sarepta \u62a5\u544a\u79f0\uff0c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 Elevidys \u57fa\u56e0\u7597\u6cd5\u4e0d\u4f1a\u7528\u4e8e\u65e0\u6cd5\u884c\u8d70\u7684\u60a3\u8005"},"content":{"rendered":"<p class=\"wp-block-paragraph\">ELEVIDYS\uff08delandistrogene moxeparvovec-rokl\uff09\u662f\u552f\u4e00\u83b7\u6279\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u57fa\u56e0\u7597\u6cd5\uff0c\u73b0\u5df2\u6536\u5230\u7f55\u89c1\u75c5\u7cbe\u51c6\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u7684\u9886\u5bfc\u8005Sarepta Therapeutics\u7684\u5b89\u5168\u6027\u66f4\u65b0\u3002\u8be5\u516c\u53f8\u8fd8\u8ba8\u8bba\u4e86\u5176\u6b63\u5728\u91c7\u53d6\u7684\u63aa\u65bd\uff0c\u4ee5\u6539\u5584\u5176\u5728\u975e\u5367\u5e8a\u60a3\u8005\u4e2d\u7684\u5b89\u5168\u6027\u3002\u5728\u53d1\u751f\u7b2c\u4e8c\u4f8b\u8bb0\u5f55\u5728\u6848\u7684\u6025\u6027\u809d\u8870\u7aed (ALF) \u5e76\u5bfc\u81f4\u6b7b\u4ea1\u7684\u75c5\u4f8b\u540e\uff0c\u91c7\u53d6\u4e86\u8fd9\u4e9b\u63aa\u65bd\u3002\u8fc4\u4eca\u4e3a\u6b62\uff0c\u4e24\u8d77ALF\u4e8b\u4ef6\u5747\u53d1\u751f\u5728\u975e\u5367\u5e8a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u8eab\u4e0a\u3002Sarepta\u5411\u53d7\u5f71\u54cd\u7684\u5bb6\u5c5e\u548c\u62a4\u7406\u4eba\u5458\u81f4\u4ee5\u6700\u6df1\u5207\u7684\u6170\u95ee\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0e\u6b64\u540c\u65f6\uff0c\u7f57\u6c0f\u516c\u53f8\u4eca\u5929\u8fd8\u516c\u5e03\u4e86ELEVIDYS\uff08delandistrogene moxeparvovec\uff09\u7684\u4fee\u8ba2\u5242\u91cf\u6307\u5357\uff0c\u8be5\u6307\u5357\u5c06\u7acb\u5373\u5bf9\u60a3\u6709\u975e\u6b65\u884c\u6027\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u7684\u60a3\u8005\u751f\u6548\uff0c\u65e0\u8bba\u5e74\u9f84\u5927\u5c0f\uff0c\u65e0\u8bba\u662f\u5728\u4e34\u5e8a\u8fd8\u662f\u5546\u4e1a\u80cc\u666f\u4e0b\u3002 <strong>Elevidys \u4e0d\u5e94\u518d\u5728\u5546\u4e1a\u73af\u5883\u4e2d\u7528\u4e8e\u975e\u5367\u5e8a\u60a3\u8005\u3002<\/strong> \u9664\u975e\u7814\u7a76\u65b9\u6848\u5305\u542b\u989d\u5916\u7684\u98ce\u9669\u7f13\u89e3\u7b56\u7565\uff08\u4f8b\u5982\u514d\u75ab\u8c03\u8282\u836f\u7269\uff09\uff0c\u5426\u5219\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u65e0\u6cd5\u884c\u8d70\u7684\u60a3\u8005\u5165\u7ec4\u548c\u7ed9\u836f\u5c06\u7acb\u5373\u505c\u6b62\u3002\u4fe1\u606f\u5c06\u4e0e\u533b\u751f\u3001\u7814\u7a76\u4eba\u5458\u548c\u536b\u751f\u5f53\u5c40\u5171\u4eab\uff0c\u4ee5\u4fbf\u53ca\u65f6\u8c03\u6574\u60a3\u8005\u62a4\u7406\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">Sarepta \u62a5\u544a\u7b2c\u4e8c\u4f8b\u57fa\u56e0\u6cbb\u7597\u540e\u809d\u8870\u7aed\u6b7b\u4ea1\u75c5\u4f8b<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e00\u51b3\u5b9a\u662f\u5728\u4ed4\u7ec6\u8bc4\u4f30\u4e86\u4e24\u4f8b\u975e\u5367\u5e8a\u60a3\u8005\u81f4\u547d\u6027\u6025\u6027\u809d\u8870\u7aed (ALF) \u75c5\u4f8b\u540e\u505a\u51fa\u7684\u3002Elevidys \u548c\u5176\u4ed6 AAV \u4ecb\u5bfc\u7684\u57fa\u56e0\u7597\u6cd5\u88ab\u53d1\u73b0\u5b58\u5728\u98ce\u9669\uff0c\u56e0\u6b64\u91cd\u65b0\u8bc4\u4f30\u4e86\u5176\u83b7\u76ca-\u98ce\u9669\u72b6\u51b5\uff0c\u8ba4\u4e3a\u5176\u5bf9\u975e\u5367\u5e8a DMD \u60a3\u8005\u4e0d\u5229\u3002\u2014\u2014\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\" target=\"_blank\" rel=\"noreferrer noopener\">Sarepta \u62a5\u544a\u79f0 16 \u5c81\u7537\u5b69\u63a5\u53d7 Elevidys \u57fa\u56e0\u6cbb\u7597\u540e\u6b7b\u4ea1<\/a> \u2013<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u65b0\u7684\u5242\u91cf\u9650\u5236\u4e0d\u4f1a\u5f71\u54cd\u4efb\u4f55\u5e74\u9f84\u7684\u95e8\u8bca DMD \u60a3\u8005\u7684\u6cbb\u7597\uff0c\u5e76\u4e14\u5728\u95e8\u8bca\u60a3\u8005\u7fa4\u4f53\u4e2d\u6548\u76ca\u98ce\u9669\u6bd4\u4ecd\u7136\u4e3a\u6b63\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e24\u4f8b\u81f4\u547d\u6027\u6025\u6027\u809d\u8870\u7aed (ALF) \u75c5\u4f8b\u5747\u53d1\u751f\u5728\u65e0\u6cd5\u884c\u8d70\u7684\u60a3\u8005\u4e2d\uff0c\u800c\u8fc4\u4eca\u4e3a\u6b62\uff0c\u5168\u7403\u5171\u6709\u7ea6140\u4f8b\u63a5\u53d7Elevidys\u6cbb\u7597\u7684\u65e0\u6cd5\u884c\u8d70\u60a3\u8005\u3002\u5728\u51fa\u73b0\u9996\u4f8b\u81f4\u547d\u6027ALF\u75c5\u4f8b\u540e\uff0c\u6b27\u6d32\u76d1\u7ba1\u673a\u6784\u8981\u6c42\u7f57\u6c0f\u548c\u8428\u96f7\u666e\u5854\u6682\u65f6\u505c\u6b62Elevidys\u7814\u7a76104 (NCT06241950)\u3001302 (ENVOL, NCT06128564) \u548c303 (ENVISION\u7814\u7a76303, NCT05310071) \u7684\u4e34\u5e8a\u7814\u7a76\u3002\u8fd9\u4e9b\u6682\u65f6\u7684\u4e34\u5e8a\u7814\u7a76\u6682\u505c\u4ecd\u5728\u8fdb\u884c\u4e2d\u3002\u5728\u6b27\u6d32\u4ee5\u5916\u7684\u5730\u533a\uff0cENVISION\u8bd5\u9a8c\u7684\u7ed9\u836f\u5c06\u7acb\u5373\u6682\u505c\u3002\u8fd9\u4e9b\u7ed9\u836f\u9650\u5236\u4e5f\u5c06\u5728\u672a\u6765\u5bf9\u5546\u4e1a\u5316\u65e0\u6cd5\u884c\u8d70\u60a3\u8005\u7ed9\u836f\u65f6\u751f\u6548\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <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>ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, has received a safety update from Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases. The company also discussed the actions it is taking to improve the safety profile in non-ambulatory patients. Following a second documented instance of acute [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3926,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[121,33,61,203,65],"class_list":["post-3925","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3925","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=3925"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3925\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3926"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3925"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3925"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3925"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}