{"id":3477,"date":"2025-04-01T17:31:55","date_gmt":"2025-04-01T14:31:55","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3477"},"modified":"2025-04-04T10:01:49","modified_gmt":"2025-04-04T07:01:49","slug":"three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/three-ongoing-clinical-trials-of-elevidys-gene-therapy-temporarily-halted-in-europe\/","title":{"rendered":"\u6b27\u6d32\u6682\u505c\u4e09\u9879\u6b63\u5728\u8fdb\u884c\u7684 Elevidys \u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c"},"content":{"rendered":"<p class=\"wp-block-paragraph\">ELEVIDYS\uff08delandistrogene moxeparvovec-rokl\uff09\u662f\u4e00\u79cd\u7531 Sarepta \u5f00\u53d1\u3001\u7f57\u6c0f\u516c\u53f8\u9500\u552e\u7684\u57fa\u56e0\u7597\u6cd5\uff0c\u76ee\u524d\u6b63\u5728\u8fdb\u884c\u7684\u4e09\u9879\u4e34\u5e8a\u8bd5\u9a8c\u5df2\u88ab\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u6682\u65f6\u53eb\u505c\uff08EMA\uff09\u3002\u8fd9\u4e00\u610f\u6599\u4e4b\u4e2d\u4f46\u5e76\u975e\u4e0d\u53d7\u6b22\u8fce\u7684\u6d88\u606f\u8ba9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u7fa4\u4f53\u6df1\u611f\u60b2\u75db\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6682\u505c\u7684\u8bd5\u9a8c\u5305\u62ec Sarepta \u8d44\u52a9\u7684 3 \u671f Envision \u7814\u7a76\uff0c\u8be5\u7814\u7a76\u6b63\u5728\u8bc4\u4f30 8 \u81f3 17 \u5c81\u7537\u5b69\u7684 Elevidys\uff1b\u7f57\u6c0f\u7684 2 \u671f Envol \u7814\u7a76\uff0c\u8be5\u7814\u7a76\u6b63\u5728\u7814\u7a76\u5a74\u513f\u548c\u65b0\u751f\u513f\u7684 Elevidys\uff1b\u4ee5\u53ca Sarepta \u7684 104 \u7814\u7a76\uff0c\u8fd9\u662f\u4e00\u9879 1 \u671f\u7814\u7a76\uff0c\u9488\u5bf9 4 \u81f3 9 \u5c81\u5df2\u5b58\u5728\u7279\u5b9a\u8840\u6e05\u578b\u6297\u4f53\u7684\u7537\u5b69\u7684 Elevidys\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728 Roche\u3001Sarepta \u548c EMA \u4e4b\u524d\uff0c\u7b2c\u4e00\u4e2a\u53d1\u5e03\u6b64\u516c\u544a\u7684\u793e\u533a\u662f\u6211\u4eec\u7684\u571f\u8033\u5176\u5408\u4f5c\u4f19\u4f34 &gt;&gt;&gt; <a href=\"https:\/\/dmd.arti.net\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD Dayanisma<\/a>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7f57\u6c0f\u516c\u53f8\u5411\u793e\u533a\u53d1\u51fa\u4e86\u4e00\u5c01\u4fe1\uff0c\u60a8\u53ef\u4ee5\u5728\u6b64\u5904\u9605\u8bfb\u3002\uff08<a href=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/March-2025-Roche-DMD-Temporary-Clinical-Study-Halt-Community-Letter_31Mar25.pdf\" target=\"_blank\" rel=\"noreferrer noopener\">PDF<\/a>)<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u76ee\u5f55<\/h2><nav><ul><li><a href=\"#why-were-elevidys-clinical-trials-halted\">Elevidys\u4e34\u5e8a\u8bd5\u9a8c\u4e3a\u4f55\u505c\u6b62\uff1f<\/a><ul><li><a href=\"#srp-9001-104\">SRP-9001-104 \u6682\u65f6\u505c\u6b62<\/a><\/li><li><a href=\"#envol-study-302\">ENVOL\uff08\u7814\u7a76 302\uff09\u6682\u65f6\u505c\u6b62<\/a><\/li><li><a href=\"#srp-9001-303\">SRP-9001-303 \u6682\u65f6\u505c\u6b62<\/a><\/li><\/ul><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-were-elevidys-clinical-trials-halted\">Elevidys\u4e34\u5e8a\u8bd5\u9a8c\u4e3a\u4f55\u505c\u6b62\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">2025\u5e743\u670818\u65e5\uff0c\u4e00\u4f4d\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u768416\u5c81\u7537\u5b69\u5728\u63a5\u53d7Elevidys\u8f93\u6ce8\u540e\u6b7b\u4ea1\uff0c\u8fd9\u4e00\u5546\u4e1a\u552e\u4ef7\u4e3a$3\u767e\u4e07\u7f8e\u5143\u7684\u57fa\u56e0\u7597\u6cd5\u7684\u524d\u666f\u7a81\u7136\u53d1\u751f\u6539\u53d8\u3002<a href=\"https:\/\/dmdwarrior.com\/zh\/sixteen-year-old-dies-after-receiving-sareptas-duchenne-gene-therapy-elevidys\/\">\u9605\u8bfb\u66f4\u591a<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e00\u4f4d\u7f8e\u56fd\u9752\u5c11\u5e74\u5728\u670d\u7528 Elevidys \u540e\u51fa\u73b0\u6025\u6027\u809d\u529f\u80fd\u8870\u7aed (ALF)\uff0c\u5728\u67e5\u660e\u5176\u786e\u5207\u6b7b\u56e0\u4e4b\u524d\uff0c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u8981\u6c42\u6682\u505c\u7814\u7a76\u3002\u4e0a\u4e2a\u6708\u60a3\u8005\u53bb\u4e16\u540e\uff0cSarepta \u5ba3\u5e03\u5c06\u4fee\u6539 Elevidys \u7684\u6807\u7b7e\uff0c\u4ee5\u52a0\u5165\u5b89\u5168\u4fe1\u53f7\u3002<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-104\">SRP-9001-104 \u6682\u65f6\u505c\u6b62<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"113\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg\" alt=\"Elevidys\u57fa\u56e0\u7597\u6cd5SRP-9001-104\u4e34\u5e8a\u8bd5\u9a8c\u5728\u6b27\u6d32\u6682\u505c\" class=\"wp-image-3480\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-300x44.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-18x3.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-150x22.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-104-HALTED-696x103.jpg 696w\" sizes=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">\u6765\u6e90\uff1a <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512624-11-00?lang=en\" target=\"_blank\" rel=\"noopener\">SRP-9001-104\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"envol-study-302\">ENVOL\uff08\u7814\u7a76 302\uff09\u6682\u65f6\u505c\u6b62<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"762\" height=\"272\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg\" alt=\"ENVOL\uff08\u7814\u7a76 302\uff09Elevidys \u57fa\u56e0\u7597\u6cd5\u4e34\u5e8a\u8bd5\u9a8c\u5728\u6b27\u6d32\u6682\u505c\" class=\"wp-image-3481\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted.jpg 762w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-300x107.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-18x6.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-150x54.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/ENVOL-Study-302-Halted-696x248.jpg 696w\" sizes=\"auto, (max-width: 762px) 100vw, 762px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">\u6765\u6e90\uff1a <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2023-509901-57-00?lang=en\" target=\"_blank\" rel=\"noopener\">SRP-9001-302\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"srp-9001-303\">SRP-9001-303 \u6682\u65f6\u505c\u6b62<\/h3>\n\n\n\n<figure class=\"wp-block-image size-full\"><img loading=\"lazy\" decoding=\"async\" width=\"768\" height=\"313\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg\" alt=\"Elevidys\u57fa\u56e0\u7597\u6cd5SRP-9001-303\u4e34\u5e8a\u8bd5\u9a8c\u5728\u6b27\u6d32\u6682\u505c\" class=\"wp-image-3482\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-300x122.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-18x7.jpg 18w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-150x61.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/04\/SRP-9001-303-Halted-696x284.jpg 696w\" sizes=\"auto, (max-width: 768px) 100vw, 768px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">\u6765\u6e90\uff1a <a href=\"https:\/\/euclinicaltrials.eu\/ctis-public\/view\/2024-512626-28-00?lang=en\" target=\"_blank\" rel=\"noopener\">SRP-9001-303 \u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-warriors-turkish-representative-shares-his-views-on-elevidys-gene-therapy-is-it-effective-and-why-is-it-expensive\/\">DMD WarrioR \u571f\u8033\u5176\u4ee3\u8868\u5206\u4eab\u4e86\u4ed6\u5bf9 Elevidys \u57fa\u56e0\u6cbb\u7597\u7684\u770b\u6cd5\u3002<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a gene therapy developed by Sarepta and marketed by Roche, have been temporarily halted by the European Medicines Agency (EMA). This expected but not unwelcome news has caused deep sadness in the Duchenne muscular dystrophy (DMD) community. The paused trials include the Sarepta-sponsored phase 3 Envision study, [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3478,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[242,33,179,91,84,213,61,203,65],"class_list":["post-3477","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-clinical-trials","tag-elevidys","tag-elevidys-gene-therapy","tag-ema","tag-europe","tag-european-medicines-agency","tag-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3477","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=3477"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3477\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3478"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3477"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3477"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3477"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}