{"id":5484,"date":"2026-01-26T16:38:21","date_gmt":"2026-01-26T13:38:21","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5484"},"modified":"2026-01-26T23:15:59","modified_gmt":"2026-01-26T20:15:59","slug":"sarepta-3-year-topline-data-of-elevidys-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/sarepta-3-year-topline-data-of-elevidys-gene-therapy\/","title":{"rendered":"Sarepta\u516c\u53f8\u516c\u5e03\u4e86ELEVIDYS\u57fa\u56e0\u7597\u6cd5\u6cbb\u7597\u53ef\u72ec\u7acb\u884c\u8d70\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u76843\u5e74\u521d\u6b65\u6570\u636e\u3002"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Sarepta \u516c\u5e03\u4e86 EMBARK\uff089001-301 \u7814\u7a76\uff09\u7b2c\u4e00\u90e8\u5206\u6cbb\u7597\u60a3\u8005\u7684 3 \u5e74\u4e3b\u8981\u529f\u80fd\u7ed3\u679c\u3002EMBARK \u662f\u4e00\u9879\u5168\u7403\u6027\u3001\u968f\u673a\u3001\u5b89\u6170\u5242\u5bf9\u7167\u7684 3 \u671f\u7814\u7a76\uff0c\u65e8\u5728\u8bc4\u4f30 ELEVIDYS\uff08delandistrogene moxeparvovec-rokl\uff09\u5bf9 4 \u81f3 7 \u5c81\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e14\u80fd\u591f\u884c\u8d70\u7684\u60a3\u8005\u7684\u7597\u6548\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Sarepta \u6ca1\u6709\u62ab\u9732 3 \u5e74\u6570\u636e\u4e2d\u7684\u808c\u9178\u6fc0\u9176\u6c34\u5e73\uff0c\u8fd9\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6700\u91cd\u8981\u7684\u751f\u7269\u6807\u5fd7\u7269\u3002.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u63a5\u53d7 ELEVIDYS \u6cbb\u7597\u7684\u60a3\u8005\u5e73\u5747\u5e74\u9f84\u4e3a 9 \u5c81\uff0c\u6cbb\u7597\u4e09\u5e74\u540e\uff0c\u5176\u5317\u6781\u661f\u6b65\u884c\u8bc4\u4f30 (NSAA) \u5e73\u5747\u5f97\u5206\u9ad8\u4e8e\u57fa\u7ebf\u6c34\u5e73 (n=52)\u3002<\/li>\n\n\n\n<li>\u4e0e\u503e\u5411\u6027\u8bc4\u5206\u52a0\u6743\u7684\u5916\u90e8\u5bf9\u7167\u7ec4\u76f8\u6bd4\uff0cELEVIDYS\u57fa\u56e0\u7597\u6cd5\u5728\u201c\u8d77\u7acb\u65f6\u95f4\u201d\uff08TTR\uff09\u548c10\u7c73\u6b65\u884c\/\u8dd1\u6b65\uff0810MWR\uff09\u6307\u6807\u4e0a\u663e\u793a\u51fa70%\u6216\u66f4\u9ad8\u7684\u8870\u9000\u7387\u964d\u4f4e\u3002\u63a5\u53d7ELEVIDYS\u6cbb\u7597\u7684\u60a3\u8005\u7597\u6548\u968f\u65f6\u95f4\u63a8\u79fb\u800c\u589e\u5f3a\uff0c\u4e0e\u5916\u90e8\u5bf9\u7167\u7ec4\u76f8\u6bd4\uff0c\u529f\u80fd\u5dee\u8ddd\u5728\u7b2c2\u5e74\u548c\u7b2c3\u5e74\u4e4b\u95f4\u663e\u8457\u6269\u5927\u3002<\/li>\n\n\n\n<li>\u672a\u89c2\u5bdf\u5230\u65b0\u7684\u4e0e\u6cbb\u7597\u76f8\u5173\u7684\u5b89\u5168\u6027\u4fe1\u53f7\uff0c\u8fd9\u4e0e\u8fc4\u4eca\u4e3a\u6b62\u5728\u95e8\u8bca\u60a3\u8005\u4e2d\u89c2\u5bdf\u5230\u7684ELEVIDYS\u53ef\u63a7\u5b89\u5168\u6027\u7279\u5f81\u4e00\u81f4\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5317\u6781\u661f\u52a8\u6001\u8bc4\u4f30\uff08NSAA\uff09<\/a><\/p>\n\n\n\n<figure class=\"wp-block-image size-full\"><img decoding=\"async\" width=\"778\" height=\"118\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png\" alt=\"Sarepta\u516c\u53f8\u516c\u5e03\u4e86ELEVIDYS\u57fa\u56e0\u7597\u6cd5\u6cbb\u7597\u53ef\u72ec\u7acb\u884c\u8d70\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u76843\u5e74\u521d\u6b65\u6570\u636e\u3002\" class=\"wp-image-5496\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table.png 778w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-300x46.png 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-768x116.png 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-18x3.png 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-150x23.png 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2026\/01\/Elevidys-EMBARK-Results-Table-696x106.png 696w\" sizes=\"(max-width: 778px) 100vw, 778px\" \/><\/figure>\n\n\n\n<figure class=\"wp-block-table\"><table class=\"has-fixed-layout\"><tbody><tr><td><strong>\u529f\u80fd\u6027\u7ed3\u679c<\/strong><\/td><td><strong>LSM \u53d8\u5316\u5dee\u5f02\u4e0e EC \u76f8\u6bd4<\/strong><\/td><td><strong>p \u503c<\/strong><\/td><\/tr><tr><td>NSAA<\/td><td>+4.39 \u5206\uff08\u63d0\u9ad8\uff09<\/td><td>p=0.0002<\/td><\/tr><tr><td>TTR<\/td><td>-6.05 \u79d2\uff08\u6539\u8fdb\uff09<\/td><td>p&lt;0.0001<\/td><\/tr><tr><td>10\u5146\u74e6<\/td><td>-2.70 \u79d2\uff08\u6539\u8fdb\uff09<\/td><td>p=0.0039<\/td><\/tr><\/tbody><\/table><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">2026\u5e741\u670826\u65e5\uff0cSarepta\u516c\u53f8\u80a1\u7968\u5f00\u76d8\u4ef7\u4e3a22\u7f8e\u5143\uff0c\u968f\u540e\u5728\u516c\u5e03\u5176Elevidys\u57fa\u56e0\u7597\u6cd5\u4e09\u5e74\u6570\u636e\u540e\u4e0a\u6da8\u81f324.68\u7f8e\u5143\u3002\u7136\u800c\uff0c\u6570\u5c0f\u65f6\u540e\uff0c\u80a1\u4ef7\u56de\u843d\uff0c\u622a\u81f3\u7f8e\u56fd\u4e1c\u90e8\u65f6\u95f4\u4e0b\u53481\u70b904\u5206\uff0c\u62a522.52\u7f8e\u5143\u3002\u6b64\u6b21\u4e0b\u8dcc\u88ab\u89e3\u8bfb\u4e3a\u6295\u8d44\u8005\u5bf9Elevidys\u57fa\u56e0\u7597\u6cd5\u4e09\u5e74\u6570\u636e\u5e76\u4e0d\u6ee1\u610f\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys\u57fa\u56e0\u6cbb\u7597\u4e2d\u7684\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u3001\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u548cNSAA\u8bc4\u5206<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Sarepta announced 3-year topline functional results from patients treated in Part One of EMBARK (Study 9001-301), the global, randomized placebo-controlled Phase 3 study evaluating ELEVIDYS (delandistrogene moxeparvovec-rokl) in ambulatory individuals with Duchenne muscular dystrophy who were aged four to seven at time of treatment. Sarepta were not disclosed creatine kinase levels, the most major biomarker [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5485,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[66,33,179,65],"class_list":["post-5484","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-duchenne-muscular-dystrophy","tag-elevidys","tag-elevidys-gene-therapy","tag-sarepta"],"subtitle":"Sarepta\u516c\u53f8\u516c\u5e03\u7684\u4e09\u5e74\u6570\u636e\u4e2d\u5e76\u672a\u62ab\u9732\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6700\u91cd\u8981\u7684\u751f\u7269\u6807\u5fd7\u7269\u2014\u2014\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u3001\u5929\u51ac\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff08AST\uff09\u548c\u4e19\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff08ALT\uff09\u3002\u8be5\u516c\u53f8\u76ee\u524d\u4ecd\u4ee5100\u4e07\u81f3290\u4e07\u7f8e\u5143\u7684\u4ef7\u683c\u5411\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u63d0\u4f9b\u5355\u5242\u91cf\u57fa\u56e0\u6cbb\u7597\u3002\u5728\u5176\u4e09\u5e74\u6570\u636e\u4e2d\uff0cSarepta\u4f3c\u4e4e\u5c06\u91cd\u70b9\u653e\u5728\u4e86NSAA\u8bc4\u5206\u4e0a\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7fa4\u4f53\u8ba4\u4e3a\u57fa\u56e0\u7597\u6cd5\u8fc7\u4e8e\u6602\u8d35\uff0c\u4e14\u73b0\u6709\u6570\u636e\u4e0d\u8db3\u3002.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5484","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=5484"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5484\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5485"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5484"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5484"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5484"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}