{"id":5141,"date":"2025-11-13T11:00:25","date_gmt":"2025-11-13T08:00:25","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5141"},"modified":"2025-11-13T11:10:04","modified_gmt":"2025-11-13T08:10:04","slug":"why-does-the-fda-approve-dmd-therapies-that-fail-in-clinical-trials","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/why-does-the-fda-approve-dmd-therapies-that-fail-in-clinical-trials\/","title":{"rendered":"\u4e3a\u4ec0\u4e48 FDA \u4f1a\u6279\u51c6\u90a3\u4e9b\u5728\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u5931\u8d25\u7684 DMD \u7597\u6cd5\uff1f"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>\u57fa\u56e0\u7597\u6cd5\uff08\u552e\u4ef7\u6570\u767e\u4e07\u7f8e\u5143\uff09\u548c\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\uff08\u6bcf\u6708\u6ce8\u5c04\u4e00\u6b21\uff0c\u552e\u4ef7\u6570\u5343\u7f8e\u5143\uff09\u771f\u7684\u6709\u6548\u5417\uff1f<\/strong> \u627f\u53d7\u7740\u5de8\u5927\u538b\u529b\u7684\u5bb6\u5ead\u6b63\u5728\u79ef\u6781\u4e89\u53d6\u83b7\u5f97\u6cbb\u7597\uff0c\u4ed6\u4eec\u5e0c\u671b DMD \u7597\u6cd5\u80fd\u591f\u5bf9\u4ed6\u4eec\u5b69\u5b50\u7684\u751f\u6d3b\u4ea7\u751f\u79ef\u6781\u5f71\u54cd\uff0c\u4f46\u6570\u636e\u5e76\u4e0d\u4e50\u89c2\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u672c\u6708\uff0cSarepta Therapeutics\u516c\u53f8\u5ba3\u5e03\uff0c\u5176\u4e24\u79cd\u5df2\u4e0a\u5e02\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5916\u663e\u5b50\u8df3\u8dc3\u836f\u7269\u7684\u9a8c\u8bc1\u6027\u8bd5\u9a8c\u672a\u80fd\u8fbe\u5230\u4e3b\u8981\u7ec8\u70b9\uff0c\u8fd9\u6216\u8bb8\u610f\u5473\u7740\u8fd9\u4e9b\u836f\u7269\u7684\u7814\u53d1\u4e4b\u8def\u5df2\u7ecf\u8d70\u5230\u4e86\u5c3d\u5934\u3002 <a href=\"https:\/\/dmdwarrior.com\/zh\/sarepta-dmd-exon-skipping-therapies-fail-confirmatory-study-stock-price-down\/\" target=\"_blank\" rel=\"noreferrer noopener\">Sarepta\u516c\u53f8DMD\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u9a8c\u8bc1\u6027\u7814\u7a76\u5931\u8d25\uff0c\u80a1\u4ef7\u4e0b\u8dcc<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u516c\u53f8\u9886\u5bfc\u5c42\u5e76\u4e0d\u613f\u610f\u653e\u5f03\u8fd9\u4e00\u60f3\u6cd5\u3002\u57fa\u4e8e\u57286\u81f313\u5c81\u7814\u7a76\u53c2\u4e0e\u8005\u8eab\u4e0a\u89c2\u5bdf\u5230\u7684\u5176\u4ed6\u5177\u6709\u4e34\u5e8a\u610f\u4e49\u7684\u53d8\u5316\uff0c\u4ed6\u4eec\u6253\u7b97\u4e0eFDA\u516c\u53f8\u4f1a\u9762\uff0c\u8ba8\u8bba\u201c\u4ece\u52a0\u901f\u5ba1\u6279\u8f6c\u4e3a\u4f20\u7edf\u5ba1\u6279\u201d\u7684\u4e8b\u5b9c\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e34\u5e8a\u8bd5\u9a8c\u5931\u8d25\u5e76\u975e\u603b\u662f\u610f\u5473\u7740\u5931\u8d25\uff0cSarepta \u4e5f\u5e76\u975e\u9996\u5bb6\u610f\u8bc6\u5230\u8fd9\u4e00\u70b9\u7684\u516c\u53f8\uff1b\u4e8b\u5b9e\u4e0a\uff0c\u5b83\u5df2\u7ecf\u633d\u6551\u4e86\u4e00\u79cd\u836f\u7269\u514d\u4e8e\u592d\u6298\u3002\u5c3d\u7ba1\u5176 DMD \u57fa\u56e0\u6cbb\u7597\u836f\u7269 Elevidys \u5728\u540e\u671f\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\u672a\u80fd\u8fbe\u5230\u4e3b\u8981\u7ec8\u70b9\uff0c\u4f46\u6700\u7ec8\u4ecd\u83b7\u5f97\u5168\u9762\u6279\u51c6\u5e76\u6269\u5927\u4e86\u9002\u5e94\u75c7\u8303\u56f4\u3002 <a href=\"https:\/\/dmdwarrior.com\/zh\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4e3a\u4ec0\u4e48Elevidys\u672a\u83b7\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\u6279\u51c6\uff1f<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u7075\u6d3b\u7684\u7b56\u7565\u4e5f\u5b58\u5728\u4e00\u4e9b\u53cd\u5bf9\u8005\u3002\u4e00\u4e9b\u4e13\u5bb6\u8ba4\u4e3a\uff0c\u5982\u679c\u836f\u7269\u8bd5\u9a8c\u5931\u8d25\uff0c\u516c\u53f8\u5e94\u8be5\u660e\u786e\u8868\u793a\u505c\u6b62\u8bd5\u9a8c\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u5728\u6211\u770b\u6765\uff0c\u8fd9\u5b8c\u5168\u6ca1\u6709\u56de\u65cb\u4f59\u5730\uff0c\u201d\u8036\u9c81\u5927\u5b66\u533b\u5b66\u9662\u533b\u5b66\u4e0e\u516c\u5171\u536b\u751f\u6559\u6388\u7ea6\u745f\u592b\u00b7\u7f57\u65af\u535a\u58eb\u8bf4\u3002\u201c\u6211\u65e0\u6cd5\u7406\u89e3\uff0c\u4e00\u9879\u65e8\u5728\u8bc1\u660e\u836f\u7269\u662f\u5426\u6709\u6548\u7684\u8bd5\u9a8c\uff0c\u5982\u679c\u5931\u8d25\u4e86\uff0c\u600e\u4e48\u8fd8\u80fd\u6279\u51c6\u8fd9\u79cd\u836f\u7269\u4e0a\u5e02\u3002\u56e0\u4e3a\u8fd9\u9879\u8bd5\u9a8c\u7684\u610f\u4e49\u4f55\u5728\uff1f\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8bd5\u9a8c\u7684\u8bbe\u8ba1\u65e8\u5728\u4e3a\u4ea7\u54c1\u63d0\u4f9b\u6700\u4f73\u673a\u4f1a\u6765\u8bc1\u660e\u5176\u76ca\u5904\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u201c\u6240\u4ee5\uff0c\u5982\u679c\u5b83\u4e3a\u7a7a\uff0c\u90a3\u5c31\u662f\u7a7a\uff0c\u201d\u4ed6\u8bf4\u3002.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u52a0\u901f\u5ba1\u6279\u9014\u5f84<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd1\u5e74\u6765\uff0cFDA\u6279\u51c6\u4e86\u5927\u91cf\u8d28\u91cf\u4f4e\u52a3\u7684\u836f\u7269\u3002\u6839\u636e\u7f57\u65af\u53c2\u4e0e\u64b0\u5199\u7684\u4e00\u9879\u7814\u7a76\uff0c\u57282018\u5e74\u81f32021\u5e74\u95f4\uff0c\u6240\u6709\u83b7\u6279\u836f\u7269\u4e2d\uff0c\u8d85\u8fc710%\u6d89\u53ca\u7684\u836f\u7269\u5728\u5173\u952e\u6027\u8bd5\u9a8c\u4e2d\u672a\u80fd\u8fbe\u5230\u81f3\u5c11\u4e00\u9879\u4e3b\u8981\u7ec8\u70b9\u3002 <a href=\"https:\/\/jamanetwork.com\/journals\/jamainternalmedicine\/fullarticle\/2801023\" target=\"_blank\" rel=\"noreferrer noopener\">\u9605\u8bfb\u6587\u7ae0<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f53\u4e00\u79cd\u836f\u7269\u5728\u6ca1\u6709\u660e\u786e\u7597\u6548\u8bc1\u636e\u7684\u60c5\u51b5\u4e0b\u83b7\u5f97\u6279\u51c6\u65f6\uff0c\u4f1a\u5f15\u53d1\u5b89\u5168\u6027\u548c\u6210\u672c\u65b9\u9762\u7684\u8bf8\u591a\u98ce\u9669\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u4ed6\u8bf4\uff1a\u201d\u8fd9\u4e9b\u4ef7\u683c\u6602\u8d35\u7684\u57fa\u56e0\u7597\u6cd5\u3001\u751f\u7269\u5236\u5242\u548c\u5176\u4ed6\u7279\u6548\u836f\u53ef\u80fd\u4f1a\u8ba9\u8bb8\u591a\u5bb6\u5ead\u7834\u4ea7\u3002\u201d \u516c\u4f17\u4e5f\u9700\u8981\u627f\u62c5\u90e8\u5206\u7ecf\u6d4e\u8d1f\u62c5\uff0c\u4f8b\u5982\u66f4\u9ad8\u7684\u4fdd\u9669\u8d39\u548c\u5176\u4ed6\u8d39\u7528\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4ed6\u8868\u793a\uff0c\u9700\u8981\u5236\u5b9a\u66f4\u4e25\u683c\u7684 FDA \u6807\u51c6\u6765\u4fdd\u62a4\u60a3\u8005\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u7f57\u65af\u8bf4\uff1a\u201d\u6211\u7684\u603b\u4f53\u611f\u89c9\u662f\uff0c\u5982\u679c\u6211\u4eec\u4f5c\u4e3a\u4e00\u4e2a\u793e\u4f1a\u8981\u5141\u8bb8\u4ea7\u54c1\u5728\u8bc1\u636e\u4e0d\u8db3\u7684\u60c5\u51b5\u4e0b\u83b7\u5f97\u6279\u51c6\uff0c\u4ee5\u4fbf\u5b83\u4eec\u80fd\u591f\u66f4\u5feb\u5730\u60e0\u53ca\u60a3\u8005\uff0c\u90a3\u4e48\u6211\u4eec\u9700\u8981\u66f4\u597d\u5730\u786e\u4fdd\u516c\u53f8\u5728\u4e0a\u5e02\u540e\u7684\u5408\u7406\u65f6\u95f4\u5185\u5b8c\u6210\u9a8c\u8bc1\u6027\u8bd5\u9a8c\uff0c\u8fd9\u6837\u6211\u4eec\u624d\u80fd\u77e5\u9053\u5b83\u4eec\u6709\u6548\uff0c\u60a3\u8005\u624d\u4e0d\u4f1a\u6d6a\u8d39\u94b1\u3002\u201d.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5173\u6ce8\u6b64\u9875\u9762<\/strong>&nbsp;&gt;&gt;&gt;&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6240\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e34\u5e8a\u8bd5\u9a8c<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Do gene therapies, marketed for millions of dollars, and exon-skipping therapies administered monthly and sold for thousands of dollars, actually work well? Families campaigning under intense stress to access the therapies hope that DMD therapies will positively impact their children&#8217;s lives, but the data is less promising. It might have been the end of the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5142,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[137,33,178,88,61,65],"class_list":["post-5141","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-clinical-trial","tag-elevidys","tag-exon-skipping-therapies","tag-fda","tag-gene-therapy","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5141","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=5141"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5141\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5142"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5141"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5141"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5141"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}