{"id":8116,"date":"2026-06-12T19:47:11","date_gmt":"2026-06-12T16:47:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8116"},"modified":"2026-06-12T20:04:35","modified_gmt":"2026-06-12T17:04:35","slug":"if-elevidys-works-why-is-sarepta-stock-falling","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/if-elevidys-works-why-is-sarepta-stock-falling\/","title":{"rendered":"\u5982\u679c Elevidys \u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6709\u6548\uff0c\u4e3a\u4ec0\u4e48 Sarepta \u7684\u80a1\u4ef7\u53cd\u800c\u4e0b\u8dcc\uff1f\u60a3\u8005\u5bb6\u5ead\u5728\u9009\u62e9\u57fa\u56e0\u7597\u6cd5\u524d\u9700\u8981\u4e86\u89e3\u54ea\u4e9b\u4fe1\u606f\uff1f"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u5f88\u5c11\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u6cbb\u7597\u65b9\u6cd5\u50cf Elevidys\uff08delandistrogene moxeparvovec\uff09\u90a3\u6837\u5f15\u8d77\u5982\u6b64\u591a\u7684\u5174\u594b\u3001\u4e89\u8bae\u548c\u8fa9\u8bba\uff0cElevidys \u662f\u7531 Sarepta Therapeutics \u5f00\u53d1\u7684\u57fa\u56e0\u7597\u6cd5\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bf9\u8bb8\u591a\u5bb6\u5ead\u6765\u8bf4\uff0cElevidys \u4ee3\u8868\u7740\u5e0c\u671b\u3002\u5b83\u662f\u9996\u4e2a\u83b7\u6279\u7684\u57fa\u56e0\u7597\u6cd5\uff0c\u65e8\u5728\u901a\u8fc7\u5c06\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u5bfc\u5165\u808c\u8089\u7ec6\u80de\uff0c\u6765\u89e3\u51b3\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6839\u672c\u9057\u4f20\u75c5\u56e0\u3002\u8be5\u7597\u6cd5\u4e8e 2023 \u5e74\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u7684\u52a0\u901f\u6279\u51c6 (FDA)\uff0c\u5e76\u4e8e 2024 \u5e74\u83b7\u5f97\u6269\u5927\u6279\u51c6\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\u4e0e\u6b64\u540c\u65f6\uff0cSarepta \u7684\u80a1\u4ef7\u5927\u5e45\u4e0b\u8dcc\uff0c\u76d1\u7ba1\u4e89\u8bae\u4e0d\u65ad\u51fa\u73b0\uff0c\u5b89\u5168\u95ee\u9898\u65e5\u76ca\u4e25\u91cd\uff0c\u8bb8\u591a\u79d1\u5b66\u5bb6\u4ecd\u5728\u4e89\u8bba\u5176\u4e34\u5e8a\u83b7\u76ca\u7684\u771f\u6b63\u7a0b\u5ea6\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u60c5\u51b5\u5f15\u53d1\u4e86\u4e00\u4e9b\u68d8\u624b\u4f46\u91cd\u8981\u7684\u95ee\u9898\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5982\u679c Elevidys \u6709\u6548\uff0c\u4e3a\u4ec0\u4e48 Sarepta \u7684\u80a1\u7968\u4f1a\u66b4\u8dcc\uff1f<\/li>\n\n\n\n<li>\u6295\u8d44\u8005\u662f\u5426\u8ba4\u4e3a\u8fd9\u79cd\u7597\u6cd5\u65e0\u6548\uff1f<\/li>\n\n\n\n<li>\u4e3a\u4ec0\u4e48\u4e34\u5e8a\u8bd5\u9a8c\u672a\u80fd\u8fbe\u5230\u4e00\u4e9b\u4e3b\u8981\u7ec8\u70b9\uff1f<\/li>\n\n\n\n<li>\u4e3a\u4ec0\u4e48 Elevidys \u83b7\u5f97\u4e86 FDA \u7684\u6279\u51c6\uff0c\u4f46\u672a\u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u7684\u6279\u51c6\uff1f<\/li>\n\n\n\n<li>\u5bb6\u5ead\u5728\u51b3\u5b9a\u662f\u5426\u63a5\u53d7\u6cbb\u7597\u524d\u5e94\u8be5\u4e86\u89e3\u54ea\u4e9b\u4fe1\u606f\uff1f<\/li>\n\n\n\n<li>\u4eca\u5929\u63a5\u53d7 Elevidys \u6cbb\u7597\u662f\u5426\u4f1a\u5f71\u54cd\u672a\u6765\u63a5\u53d7\u5176\u4ed6\u7597\u6cd5\u7684\u8d44\u683c\uff1f<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u95ee\u9898\u9700\u8981\u4ee5\u4e8b\u5b9e\u4e3a\u4f9d\u636e\u7684\u7b54\u6848\uff0c\u800c\u4e0d\u662f\u5728\u793e\u4ea4\u5a92\u4f53\u4e0a\u8fdb\u884c\u60c5\u7eea\u5316\u7684\u4e89\u8bba\u3002.<\/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=\"#understanding-what-elevidys-actually-does\">\u4e86\u89e3 Elevidys \u7684\u5b9e\u9645\u529f\u80fd<\/a><ul><\/ul><\/li><li><a href=\"#if-elevidys-works-why-has-sareptas-stock-fallen-so-dramatically\">\u5982\u679c Elevidys \u6709\u6548\uff0c\u4e3a\u4ec0\u4e48 Sarepta \u7684\u80a1\u4ef7\u4f1a\u5927\u5e45\u4e0b\u8dcc\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#why-viral-videos-should-not-be-used-as-scientific-evidence\">\u4e3a\u4ec0\u4e48\u4e0d\u5e94\u5c06\u75c5\u6bd2\u5f0f\u4f20\u64ad\u7684\u89c6\u9891\u7528\u4f5c\u79d1\u5b66\u8bc1\u636e<\/a><ul><\/ul><\/li><li><a href=\"#are-nsaa-scores-enough-to-measure-success\">NSAA \u5206\u6570\u8db3\u4ee5\u8861\u91cf\u6210\u529f\u5417\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#how-many-extra-years-could-elevidys-add\">\u6211\u7684\u513f\u5b50\u63a5\u53d7 Elevidys \u6cbb\u7597\u540e\u8fd8\u80fd\u6d3b\u591a\u5c11\u5e74\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#is-elevidys-worth-3-2-million\">Elevidys \u503c 320 \u4e07\u7f8e\u5143\u5417\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#why-is-elevidys-approved-by-the-fda-but-not-by-the-ema\">\u4e3a\u4ec0\u4e48 Elevidys \u83b7\u5f97\u4e86 FDA \u7684\u6279\u51c6\uff0c\u5374\u6ca1\u6709\u83b7\u5f97 EMA \u7684\u6279\u51c6\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#could-receiving-elevidys-limit-future-treatment-options\">\u63a5\u53d7 Elevidys \u6cbb\u7597\u662f\u5426\u4f1a\u9650\u5236\u672a\u6765\u7684\u6cbb\u7597\u9009\u62e9\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#what-new-duchenne-therapies-are-coming\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6709\u54ea\u4e9b\u65b0\u7597\u6cd5\u5373\u5c06\u95ee\u4e16\uff1f<\/a><ul><\/ul><\/li><li><a href=\"#why-families-continue-campaigning-for-elevidys\">\u4e3a\u4ec0\u4e48\u5bb6\u5ead\u7ee7\u7eed\u4e3a Elevidys \u53d1\u8d77\u8fd0\u52a8<\/a><\/li><li><a href=\"#questions-every-family-should-ask-before-choosing-elevidys\">\u6bcf\u4e2a\u5bb6\u5ead\u5728\u9009\u62e9 Elevidys \u4e4b\u524d\u90fd\u5e94\u8be5\u95ee\u7684\u95ee\u9898<\/a><\/li><li><a href=\"#conclusion\">\u7ed3\u8bba<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"understanding-what-elevidys-actually-does\" class=\"wp-block-heading\">\u4e86\u89e3 Elevidys \u7684\u5b9e\u9645\u529f\u80fd<\/h2>\n\n\n\n<h3 id=\"elevidys-is-not-a-full-dystrophin-replacement\" class=\"wp-block-heading\">Elevidys \u5e76\u975e\u5b8c\u5168\u66ff\u4ee3\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u53d1\u751f\u662f\u56e0\u4e3a DMD \u57fa\u56e0\u7a81\u53d8\u963b\u6b62\u4e86\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\uff0c\u800c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u662f\u4e00\u79cd\u4fdd\u62a4\u808c\u8089\u7ea4\u7ef4\u514d\u53d7\u635f\u4f24\u7684\u5173\u952e\u86cb\u767d\u8d28\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys \u4e0d\u80fd\u5b8c\u5168\u6062\u590d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5b83\u5e76\u975e\u4f7f\u7528\u5b8c\u6574\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\uff0c\u800c\u662f\u5229\u7528\u817a\u76f8\u5173\u75c5\u6bd2\uff08AAV\uff09\u8f7d\u4f53\u9012\u9001\u4e00\u79cd\u540d\u4e3a\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u77ed\u7248\u672c\u3002\u4e4b\u6240\u4ee5\u8bbe\u8ba1\u8fd9\u79cd\u7597\u6cd5\uff0c\u662f\u56e0\u4e3a\u5b8c\u6574\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u592a\u5927\uff0c\u65e0\u6cd5\u88c5\u5165\u73b0\u6709\u7684AAV\u9012\u9001\u7cfb\u7edf\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u533a\u522b\u5f88\u91cd\u8981\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7597\u6cd5\u65e8\u5728\u4ea7\u751f\u4e00\u79cd\u8f83\u5c0f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6837\u86cb\u767d\uff0c\u8fd9\u79cd\u86cb\u767d\u53ef\u80fd\u90e8\u5206\u4fdd\u62a4\u808c\u8089\u7ec6\u80de\uff0c\u4f46\u5e76\u4e0d\u80fd\u5b8c\u5168\u590d\u5236\u5065\u5eb7\u4e2a\u4f53\u4f53\u5185\u5b58\u5728\u7684\u5929\u7136\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u7814\u7a76\u8868\u660e\uff0c\u6cbb\u7597\u540e\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u80fd\u591f\u6301\u7eed\u5b58\u5728\uff0c\u8fd9\u4e5f\u662fFDA\u83b7\u6279\u7684\u5173\u952e\u4f9d\u636e\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"if-elevidys-works-why-has-sareptas-stock-fallen-so-dramatically\" class=\"wp-block-heading\">\u5982\u679c Elevidys \u6709\u6548\uff0c\u4e3a\u4ec0\u4e48 Sarepta \u7684\u80a1\u4ef7\u4f1a\u5927\u5e45\u4e0b\u8dcc\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8bb8\u591a\u5bb6\u5ead\u8ba4\u4e3a\u80a1\u4ef7\u4e0b\u8dcc\u5c31\u4e00\u5b9a\u610f\u5473\u7740\u6cbb\u7597\u65e0\u6548\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u751f\u7269\u6280\u672f\u9886\u57df\uff0c\u5b9e\u9645\u60c5\u51b5\u8981\u590d\u6742\u5f97\u591a\u3002.<\/p>\n\n\n\n<h3 id=\"investor-concerns-extend-beyond-clinical-results\" class=\"wp-block-heading\">\u6295\u8d44\u8005\u62c5\u5fe7\u7684\u7126\u70b9\u4e0d\u4ec5\u9650\u4e8e\u4e34\u5e8a\u7ed3\u679c<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u6295\u8d44\u8005\u8bc4\u4f30\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5b89\u5168\u98ce\u9669<\/li>\n\n\n\n<li>\u76d1\u7ba1\u4e0d\u786e\u5b9a\u6027<\/li>\n\n\n\n<li>\u5546\u4e1a\u91c7\u7528<\/li>\n\n\n\n<li>\u4fdd\u9669\u62a5\u9500<\/li>\n\n\n\n<li>\u672a\u6765\u7ade\u4e89<\/li>\n\n\n\n<li>\u957f\u671f\u7597\u6548<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u800c\u4e0d\u662f\u4ec5\u4ec5\u8be2\u95ee\u67d0\u79cd\u6cbb\u7597\u65b9\u6cd5\u662f\u5426\u6709\u6548\u3002.<\/p>\n\n\n\n<h3 id=\"safety-concerns-have-had-major-impact\" class=\"wp-block-heading\">\u5b89\u5168\u9690\u60a3\u4ea7\u751f\u4e86\u91cd\u5927\u5f71\u54cd<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">2025 \u5e74\uff0c\u4e0e\u57fa\u56e0\u6cbb\u7597\u9879\u76ee\u76f8\u5173\u7684\u6025\u6027\u809d\u8870\u7aed\u75c5\u4f8b\u548c\u60a3\u8005\u6b7b\u4ea1\u4e8b\u4ef6\uff0c\u52a0\u5267\u4e86\u76d1\u7ba1\u673a\u6784\u548c\u6295\u8d44\u8005\u7684\u5173\u6ce8\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u540e\u6765\u66f4\u65b0\u4e86 Elevidys \u7684\u5904\u65b9\u4fe1\u606f\uff0c\u5e76\u589e\u52a0\u4e86\u5173\u4e8e\u6025\u6027\u4e25\u91cd\u809d\u635f\u4f24\u548c\u6025\u6027\u809d\u8870\u7aed\u7684\u66f4\u5f3a\u70c8\u7684\u5b89\u5168\u8b66\u544a\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u867d\u7136\u8fd9\u4e9b\u4e8b\u4ef6\u4e3b\u8981\u53d1\u751f\u5728\u65e0\u6cd5\u884c\u8d70\u7684\u60a3\u8005\u8eab\u4e0a\uff0c\u4f46\u5b83\u4eec\u5bf9\u6295\u8d44\u8005\u4fe1\u5fc3\u4ea7\u751f\u4e86\u91cd\u5927\u5f71\u54cd\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6295\u8d44\u8005\u4e0d\u4ec5\u62c5\u5fc3\u67d0\u79cd\u7597\u6cd5\u662f\u5426\u6709\u6548\uff0c\u8fd8\u62c5\u5fc3\u76d1\u7ba1\u673a\u6784\u672a\u6765\u662f\u5426\u4f1a\u9650\u5236\u5176\u4f7f\u7528\u3002.<\/p>\n\n\n\n<h3 id=\"clinical-trial-results-have-been-interpreted-differently\" class=\"wp-block-heading\">\u4e34\u5e8a\u8bd5\u9a8c\u7ed3\u679c\u7684\u89e3\u8bfb\u5404\u4e0d\u76f8\u540c<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e89\u8bae\u7684\u7126\u70b9\u4e4b\u4e00\u662f EMBARK \u7b2c\u4e09\u9636\u6bb5\u7814\u7a76\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6700\u521d\u7684\u5b89\u6170\u5242\u5bf9\u7167 EMBARK \u8bd5\u9a8c\u672a\u80fd\u8fbe\u5230\u5176\u4e3b\u8981\u7ec8\u70b9\uff0c\u5373\u4e0e\u5b89\u6170\u5242\u76f8\u6bd4\uff0c\u5317\u6781\u661f\u6b65\u884c\u8bc4\u4f30 (NSAA) \u8bc4\u5206\u7684\u6539\u5584\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u591a\u4e2a\u6b21\u8981\u7ec8\u70b9\u6307\u6807\u90fd\u5bf9 Elevidys \u6709\u5229\uff0c\u540e\u6765\u7684\u5206\u6790\u8868\u660e\u75be\u75c5\u8fdb\u5c55\u6709\u6240\u51cf\u7f13\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u652f\u6301\u8005\u8ba4\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8fdb\u5c55\u7f13\u6162\uff0c\u96be\u4ee5\u5728\u77ed\u65f6\u95f4\u5185\u8fdb\u884c\u8861\u91cf\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6279\u8bc4\u4eba\u58eb\u8ba4\u4e3a\uff0c\u672a\u80fd\u8fbe\u5230\u4e3b\u8981\u7ec8\u70b9\u5e94\u8be5\u4f1a\u5f15\u53d1\u4eba\u4eec\u5bf9\u7597\u6548\u7684\u8d28\u7591\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7ed3\u679c\u662f\u79d1\u5b66\u754c\u5b58\u5728\u5206\u6b67\uff0c\u800c\u4e0d\u662f\u8fbe\u6210\u5171\u8bc6\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-viral-videos-should-not-be-used-as-scientific-evidence\" class=\"wp-block-heading\">\u4e3a\u4ec0\u4e48\u4e0d\u5e94\u5c06\u75c5\u6bd2\u5f0f\u4f20\u64ad\u7684\u89c6\u9891\u7528\u4f5c\u79d1\u5b66\u8bc1\u636e<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bb6\u5c5e\u7ecf\u5e38\u4f1a\u5728\u793e\u4ea4\u5a92\u4f53\u4e0a\u770b\u5230\u4e00\u4e9b\u4ee4\u4eba\u9707\u60ca\u7684\u89c6\u9891\uff0c\u5c55\u793a\u5b69\u5b50\u5728\u6cbb\u7597\u524d\u540e\u7684\u5bf9\u6bd4\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u89c6\u9891\u80fd\u5f15\u53d1\u5f3a\u70c8\u7684\u60c5\u611f\u5171\u9e23\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u5b83\u4eec\u5e76\u975e\u79d1\u5b66\u8bc1\u636e\u3002.<\/p>\n\n\n\n<h3 id=\"the-problem-with-anecdotal-evidence\" class=\"wp-block-heading\">\u8f76\u4e8b\u8bc1\u636e\u7684\u95ee\u9898<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e00\u4e2a\u5b69\u5b50\u53ef\u80fd\u4f1a\u51fa\u73b0\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u66f4\u5f3a<\/li>\n\n\n\n<li>\u5feb\u70b9<\/li>\n\n\n\n<li>\u66f4\u6709\u6d3b\u529b<\/li>\n\n\n\n<li>\u66f4\u7a33\u5b9a<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u51fa\u4e8e\u8bb8\u591a\u4e0e\u6cbb\u7597\u65e0\u5173\u7684\u539f\u56e0\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f71\u54cd\u56e0\u7d20\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u751f\u957f<\/li>\n\n\n\n<li>\u7406\u7597<\/li>\n\n\n\n<li>\u76ae\u8d28\u7c7b\u56fa\u9187\u7684\u4f7f\u7528<\/li>\n\n\n\n<li>\u52a8\u673a<\/li>\n\n\n\n<li>\u81ea\u7136\u53d8\u5f02\u6027<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e00\u6bb5\u77ed\u89c6\u9891\u65e0\u6cd5\u786e\u5b9a\u67d0\u79cd\u6cbb\u7597\u65b9\u6cd5\u662f\u5426\u80fd\u6539\u53d8\u75be\u75c5\u7684\u8fdb\u5c55\u3002.<\/p>\n\n\n\n<h3 id=\"objective-biomarkers-matter-more\" class=\"wp-block-heading\">\u5ba2\u89c2\u751f\u7269\u6807\u5fd7\u7269\u66f4\u4e3a\u91cd\u8981<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bb6\u5c5e\u5e94\u8981\u6c42\u516c\u5f00\u900f\u660e\u5730\u62a5\u544a\u4ee5\u4e0b\u4fe1\u606f\uff1a<\/p>\n\n\n\n<h4 id=\"ck-creatine-kinase\" class=\"wp-block-heading\">CK\uff08\u808c\u9178\u6fc0\u9176\uff09<\/h4>\n\n\n\n<p class=\"wp-block-paragraph\">CK\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6700\u91cd\u8981\u7684\u751f\u7269\u6807\u5fd7\u7269\u4e4b\u4e00\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f53\u808c\u8089\u7ec6\u80de\u53d7\u635f\u65f6\uff0c\u808c\u9178\u6fc0\u9176\u4f1a\u6e17\u5165\u8840\u6db2\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6cbb\u7597\u540e CK \u6c34\u5e73\u964d\u4f4e\u53ef\u80fd\u8868\u660e\u808c\u8089\u635f\u4f24\u51cf\u8f7b\u3002.<\/p>\n\n\n\n<h4 id=\"ast-and-alt\" class=\"wp-block-heading\">AST \u548c ALT<\/h4>\n\n\n\n<p class=\"wp-block-paragraph\">AST \u548c ALT \u901a\u5e38\u88ab\u89c6\u4e3a\u809d\u9176\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0cAST \u548c ALT \u5347\u9ad8\u4e5f\u53ef\u80fd\u6e90\u4e8e\u53d7\u635f\u7684\u808c\u8089\u7ec4\u7ec7\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u957f\u671f\u8ffd\u8e2a\u8fd9\u4e9b\u6307\u6807\u53ef\u4ee5\u4e3a\u808c\u8089\u5065\u5eb7\u548c\u6cbb\u7597\u5b89\u5168\u6027\u63d0\u4f9b\u91cd\u8981\u7684\u89c1\u89e3\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c Elevidys \u771f\u7684\u80fd\u4fdd\u62a4\u808c\u8089\u7ea4\u7ef4\uff0c\u8bb8\u591a\u4e13\u5bb6\u8ba4\u4e3a CK \u7684\u53d8\u5316\u8d8b\u52bf\u5e94\u8be5\u4e0e\u529f\u80fd\u6027\u7ed3\u679c\u4e00\u6837\u53d7\u5230\u5173\u6ce8\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/ck-levels-after-duchenne-gene-therapy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4e3a\u4ec0\u4e48\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u4e2d\uff0cCK\u3001AST \u548c ALT \u6570\u636e\u6bd4\u89c6\u9891\u66f4\u91cd\u8981<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"are-nsaa-scores-enough-to-measure-success\" class=\"wp-block-heading\">NSAA \u5206\u6570\u8db3\u4ee5\u8861\u91cf\u6210\u529f\u5417\uff1f<\/h2>\n\n\n\n<h3 id=\"what-is-nsaa\" class=\"wp-block-heading\">\u4ec0\u4e48\u662fNSAA\uff1f<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5317\u6781\u661f\u52a8\u6001\u8bc4\u4f30\u5e7f\u6cdb\u5e94\u7528\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5b83\u8bc4\u4f30\u7684\u6d3b\u52a8\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5e38\u8bbe<\/li>\n\n\n\n<li>\u6b65\u884c<\/li>\n\n\n\n<li>\u6500\u767b<\/li>\n\n\n\n<li>\u8df3<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5206\u6570\u8d8a\u9ad8\uff0c\u8868\u660e\u8fd0\u52a8\u529f\u80fd\u8d8a\u597d\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"4959\" rel=\"noreferrer noopener\">NSAA\u662f\u4ec0\u4e48\uff1f<\/a><\/p>\n\n\n\n<h3 id=\"the-limitations-of-nsaa\" class=\"wp-block-heading\">NSAA\u7684\u5c40\u9650\u6027<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">NSAA\u53ef\u80fd\u53d7\u5230\u4ee5\u4e0b\u56e0\u7d20\u7684\u5f71\u54cd\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5e74\u9f84<\/li>\n\n\n\n<li>\u52a8\u673a<\/li>\n\n\n\n<li>\u75b2\u52b3<\/li>\n\n\n\n<li>\u8bc4\u4f30\u8005\u5dee\u5f02<\/li>\n\n\n\n<li>\u5b66\u4e60\u6548\u679c<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u513f\u7ae5\u75c5\u60c5\u53ef\u80fd\u4f1a\u6682\u65f6\u597d\u8f6c\uff0c\u4f46\u5e76\u672a\u51fa\u73b0\u5b9e\u8d28\u6027\u7684\u751f\u7406\u6539\u5584\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u56e0\u6b64\uff0c\u8bb8\u591a\u7814\u7a76\u4eba\u5458\u8ba4\u4e3a\u5e94\u8be5\u5c06 NSAA \u4e0e\u751f\u7269\u6807\u5fd7\u7269\u4e00\u8d77\u89e3\u8bfb\u3002.<\/p>\n\n\n\n<h3 id=\"why-dystrophin-production-matters\" class=\"wp-block-heading\">\u4e3a\u4ec0\u4e48\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u81f3\u5173\u91cd\u8981<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f52\u6839\u7ed3\u5e95\uff0c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u662f\u7531\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7f3a\u4e4f\u5f15\u8d77\u7684\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7406\u60f3\u60c5\u51b5\u4e0b\uff0c\u65e8\u5728\u66ff\u4ee3\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u7597\u6cd5\u5e94\u5177\u5907\u4ee5\u4e0b\u7279\u70b9\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5f3a\u52b2\u7684\u86cb\u767d\u8d28\u751f\u4ea7<\/li>\n\n\n\n<li>\u6301\u7eed\u8868\u8fbe<\/li>\n\n\n\n<li>\u808c\u8089\u4fdd\u5b58<\/li>\n\n\n\n<li>\u957f\u671f\u529f\u80fd\u6027\u83b7\u76ca<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u662f FDA \u5ba1\u7a3f\u4eba\u8ba4\u4e3a Elevidys \u5177\u6709\u751f\u7269\u6d3b\u6027\u7684\u4e3b\u8981\u539f\u56e0\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u79d1\u5b66\u5bb6\u4eec\u4ecd\u5728\u4e89\u8bba\u9700\u8981\u591a\u5c11\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u624d\u80fd\u4ea7\u751f\u6709\u610f\u4e49\u7684\u4e34\u5e8a\u76ca\u5904\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5355\u9760NSAA\u662f\u4e0d\u591f\u7684<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"how-many-extra-years-could-elevidys-add\" class=\"wp-block-heading\">\u6211\u7684\u513f\u5b50\u63a5\u53d7 Elevidys \u6cbb\u7597\u540e\u8fd8\u80fd\u6d3b\u591a\u5c11\u5e74\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u6216\u8bb8\u662f\u5bb6\u4eba\u4eec\u6700\u5173\u5fc3\u7684\u95ee\u9898\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f88\u9057\u61be\uff0c\u6ca1\u6709\u4eba\u77e5\u9053\u3002.<\/p>\n\n\n\n<h3 id=\"no-survival-benefit-has-yet-been-proven\" class=\"wp-block-heading\">\u5c1a\u672a\u8bc1\u5b9e\u5177\u6709\u751f\u5b58\u76ca\u5904<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys \u5b58\u5728\u7684\u65f6\u95f4\u8fd8\u4e0d\u591f\u957f\uff0c\u65e0\u6cd5\u8bc1\u660e\u5b83\u662f\u5426\u80fd\u901a\u8fc7\u4ee5\u4e0b\u65b9\u5f0f\u5ef6\u957f\u9884\u671f\u5bff\u547d\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>2\u5e74<\/li>\n\n\n\n<li>5\u5e74<\/li>\n\n\n\n<li>10\u5e74<\/li>\n\n\n\n<li>20\u5e74<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\u8fd8\u6ca1\u6709\u957f\u671f\u751f\u5b58\u7814\u7a76\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\u4efb\u4f55\u5173\u4e8e\u5bff\u547d\u5ef6\u957f\u7684\u5177\u4f53\u8bf4\u6cd5\u90fd\u53ea\u662f\u63a8\u6d4b\u3002.<\/p>\n\n\n\n<h3 id=\"what-clinical-trials-suggest\" class=\"wp-block-heading\">\u4e34\u5e8a\u8bd5\u9a8c\u7ed3\u679c\u63d0\u793a<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd1\u671f\u540e\u7eed\u5206\u6790\u8868\u660e\uff0c\u4e0e\u81ea\u7136\u53f2\u961f\u5217\u76f8\u6bd4\uff0cElevidys \u53ef\u80fd\u51cf\u7f13\u75be\u75c5\u8fdb\u5c55\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c\u75c5\u60c5\u8fdb\u5c55\u51cf\u7f13\u7684\u60c5\u51b5\u5728\u8f83\u957f\u65f6\u95f4\u5185\u5f97\u5230\u8bc1\u5b9e\uff0c\u7406\u8bba\u4e0a\u53ef\u4ee5\u5ef6\u7f13\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u4e27\u5931\u884c\u8d70\u80fd\u529b<\/li>\n\n\n\n<li>\u547c\u5438\u529f\u80fd\u8870\u9000<\/li>\n\n\n\n<li>\u5fc3\u810f\u5e76\u53d1\u75c7<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u8981\u8bc1\u660e\u751f\u5b58\u671f\u5ef6\u957f\uff0c\u8fd8\u9700\u8981\u591a\u5e74\u7684\u89c2\u5bdf\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bb6\u5c5e\u5e94\u8be5\u660e\u767d\uff0c\u76ee\u524d\u7684\u8bc1\u636e\u53ea\u8bc1\u660e\u4e86\u5176\u751f\u7269\u6d3b\u6027\u548c\u6f5c\u5728\u7684\u529f\u80fd\u76ca\u5904\uff0c\u800c\u6ca1\u6709\u8bc1\u660e\u5176\u80fd\u591f\u5ef6\u957f\u5bff\u547d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/elevidys-gene-therapy-efficacy-in-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys\u57fa\u56e0\u7597\u6cd5\u80fd\u5c06DMD\u60a3\u513f\u7684\u75c5\u60c5\u6539\u5584\u5230\u4ec0\u4e48\u7a0b\u5ea6\uff1f<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"is-elevidys-worth-3-2-million\" class=\"wp-block-heading\">Elevidys \u503c 320 \u4e07\u7f8e\u5143\u5417\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u636e\u62a5\u9053\uff0cElevidys \u7684\u6cbb\u7597\u8d39\u7528\u7ea6\u4e3a\u6bcf\u6b21 320 \u4e07\u7f8e\u5143\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4f7f\u5176\u6210\u4e3a\u5386\u53f2\u4e0a\u6700\u6602\u8d35\u7684\u836f\u7269\u4e4b\u4e00\u3002.<\/p>\n\n\n\n<h3 id=\"the-economic-question\" class=\"wp-block-heading\">\u7ecf\u6d4e\u95ee\u9898<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4ef7\u503c\u53d6\u51b3\u4e8e\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6536\u76ca\u7a0b\u5ea6<\/li>\n\n\n\n<li>\u798f\u5229\u6301\u7eed\u65f6\u95f4<\/li>\n\n\n\n<li>\u5b89\u5168\u6982\u51b5<\/li>\n\n\n\n<li>\u672a\u6765\u6cbb\u7597\u65b9\u6848<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c\u4e00\u79cd\u7597\u6cd5\u80fd\u5ef6\u957f\u6570\u5341\u5e74\u7684\u5065\u5eb7\u5bff\u547d\uff0c\u8bb8\u591a\u4eba\u4f1a\u8ba4\u4e3a\u5b83\u662f\u503c\u5f97\u7684\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c\u6536\u76ca\u4e0d\u5927\uff0c\u90a3\u4e48\u5176\u4ef7\u503c\u4e3b\u5f20\u5c31\u4f1a\u66f4\u5177\u4e89\u8bae\u6027\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u76ee\u524d\uff0c\u771f\u6b63\u7684\u957f\u671f\u4ef7\u503c\u4ecd\u4e0d\u786e\u5b9a\uff0c\u56e0\u4e3a\u957f\u671f\u7ed3\u679c\u4ecd\u7136\u672a\u77e5\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-is-elevidys-approved-by-the-fda-but-not-by-the-ema\" class=\"wp-block-heading\">\u4e3a\u4ec0\u4e48 Elevidys \u83b7\u5f97\u4e86 FDA \u7684\u6279\u51c6\uff0c\u5374\u6ca1\u6709\u83b7\u5f97 EMA \u7684\u6279\u51c6\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8bb8\u591a\u5bb6\u5ead\u8ba4\u4e3a\uff0c\u83b7\u5f97\u4e00\u4e2a\u76d1\u7ba1\u673a\u6784\u7684\u6279\u51c6\u5c31\u80fd\u4fdd\u8bc1\u83b7\u5f97\u5176\u4ed6\u76d1\u7ba1\u673a\u6784\u7684\u6279\u51c6\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u60c5\u51b5\u5e76\u975e\u603b\u662f\u5982\u6b64\u3002.<\/p>\n\n\n\n<h3 id=\"different-regulatory-standards\" class=\"wp-block-heading\">\u4e0d\u540c\u7684\u76d1\u7ba1\u6807\u51c6<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u548c EMA \u53ef\u80fd\u8bc4\u4f30\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u98ce\u9669\u627f\u53d7\u80fd\u529b<\/li>\n\n\n\n<li>\u4e34\u5e8a\u7ec8\u70b9<\/li>\n\n\n\n<li>\u7edf\u8ba1\u8bc1\u636e<\/li>\n\n\n\n<li>\u672a\u6ee1\u8db3\u7684\u533b\u7597\u9700\u6c42<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0d\u4e00\u6837\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u5386\u6765\u8868\u73b0\u51fa\u613f\u610f\u5229\u7528\u52a0\u901f\u5ba1\u6279\u9014\u5f84\u6cbb\u7597\u7f3a\u4e4f\u6cbb\u7597\u65b9\u6848\u7684\u4e25\u91cd\u75be\u75c5\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6b27\u6d32\u76d1\u7ba1\u673a\u6784\u6709\u65f6\u8981\u6c42\u63d0\u4f9b\u66f4\u6709\u529b\u7684\u4f50\u8bc1\u8bc1\u636e\u624d\u80fd\u6279\u51c6\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u5e76\u4e0d\u4e00\u5b9a\u610f\u5473\u7740\u5176\u4e2d\u4e00\u4e2a\u673a\u6784\u662f\u5bf9\u7684\uff0c\u800c\u53e6\u4e00\u4e2a\u673a\u6784\u662f\u9519\u7684\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5b83\u53cd\u6620\u4e86\u5bf9\u73b0\u6709\u8bc1\u636e\u7684\u4e0d\u540c\u89e3\u8bfb\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <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<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"could-receiving-elevidys-limit-future-treatment-options\" class=\"wp-block-heading\">\u63a5\u53d7 Elevidys \u6cbb\u7597\u662f\u5426\u4f1a\u9650\u5236\u672a\u6765\u7684\u6cbb\u7597\u9009\u62e9\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e2a\u95ee\u9898\u503c\u5f97\u8fdb\u884c\u6bd4\u73b0\u5728\u66f4\u591a\u7684\u8ba8\u8bba\u3002.<\/p>\n\n\n\n<h3 id=\"the-aav-antibody-problem\" class=\"wp-block-heading\">AAV\u6297\u4f53\u95ee\u9898<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys \u4f7f\u7528 AAV \u8f7d\u4f53\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6cbb\u7597\u540e\uff0c\u514d\u75ab\u7cfb\u7edf\u53ef\u80fd\u4f1a\u4ea7\u751f\u9488\u5bf9\u8be5\u8f7d\u4f53\u7684\u6297\u4f53\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u7ed9\u672a\u6765\u7684\u518d\u6b21\u7ed9\u836f\u5e26\u6765\u4e86\u6311\u6218\u3002.<\/p>\n\n\n\n<h3 id=\"potential-impact-on-future-gene-therapies\" class=\"wp-block-heading\">\u5bf9\u672a\u6765\u57fa\u56e0\u7597\u6cd5\u7684\u6f5c\u5728\u5f71\u54cd<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u8bb8\u591a\u65b0\u4e00\u4ee3\u57fa\u56e0\u7597\u6cd5\u4e5f\u4f9d\u8d56\u4e8eAAV\u6280\u672f\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bb6\u5c5e\u5e94\u8be5\u95ee\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u672a\u6765\u7684\u6cbb\u7597\u65b9\u6cd5\u4f1a\u4f7f\u7528\u7c7b\u4f3c\u7684\u8f7d\u4f53\u5417\uff1f<\/li>\n\n\n\n<li>\u6211\u8fd8\u80fd\u7b26\u5408\u8d44\u683c\u5417\uff1f<\/li>\n\n\n\n<li>\u662f\u5426\u6709\u6b63\u5728\u5236\u5b9a\u91cd\u590d\u7ed9\u836f\u7b56\u7565\uff1f<\/li>\n\n\n\n<li>\u53ef\u80fd\u4f1a\u51fa\u73b0\u54ea\u4e9b\u66ff\u4ee3\u65b9\u6848\uff1f<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u8ba8\u8bba\u5e94\u8be5\u5728\u6cbb\u7597\u524d\u8fdb\u884c\uff0c\u800c\u4e0d\u662f\u6cbb\u7597\u540e\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"what-new-duchenne-therapies-are-coming\" class=\"wp-block-heading\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6709\u54ea\u4e9b\u65b0\u7597\u6cd5\u5373\u5c06\u95ee\u4e16\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u675c\u6c0f\u8f93\u6cb9\u7ba1\u9053\u6b63\u5728\u5feb\u901f\u53d1\u5c55\u3002.<\/p>\n\n\n\n<h3 id=\"next-generation-gene-therapies\" class=\"wp-block-heading\">\u4e0b\u4e00\u4ee3\u57fa\u56e0\u7597\u6cd5<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u591a\u5bb6\u516c\u53f8\u6b63\u5728\u7814\u53d1\u6539\u8fdb\u65b9\u6cd5\uff0c\u65e8\u5728\u63d0\u9ad8\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u6216\u63d0\u5347\u5b89\u5168\u6027\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/new-gene-therapies-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6f5c\u5728\u7684\u65b0\u578b\u57fa\u56e0\u7597\u6cd5<\/a><\/p>\n\n\n\n<h3 id=\"cell-therapies\" class=\"wp-block-heading\">\u7ec6\u80de\u7597\u6cd5<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u7814\u7a76\u4eba\u5458\u6b63\u5728\u63a2\u7d22\u53ef\u80fd\u518d\u751f\u53d7\u635f\u808c\u8089\u7ec4\u7ec7\u7684\u7ec6\u80de\u7597\u6cd5\u3002.<\/p>\n\n\n\n<h3 id=\"m-rna-therapies\" class=\"wp-block-heading\">mRNA\u7597\u6cd5<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u65b0\u5174\u7684 mRNA \u65b9\u6cd5\u65e8\u5728\u751f\u4ea7\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u800c\u4e0d\u662f\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7b56\u7565\u5f15\u8d77\u4e86\u5e7f\u6cdb\u5173\u6ce8\uff0c\u56e0\u4e3a\u5b83\u53ef\u80fd\u514b\u670d\u4e0e AAV \u8f7d\u4f53\u76f8\u5173\u7684\u5c3a\u5bf8\u9650\u5236\u3002.<\/p>\n\n\n\n<h3 id=\"gene-editing\" class=\"wp-block-heading\">\u57fa\u56e0\u7f16\u8f91<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u57fa\u4e8e CRISPR \u7684\u65b9\u6cd5\u4ecd\u5904\u4e8e\u5b9e\u9a8c\u9636\u6bb5\uff0c\u4f46\u63d0\u4f9b\u4e86\u6c38\u4e45\u6027\u57fa\u56e0\u77eb\u6b63\u7684\u53ef\u80fd\u6027\u3002.<\/p>\n\n\n\n<h3 id=\"full-length-dystrophin-strategies\" class=\"wp-block-heading\">\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7b56\u7565<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u8bb8\u591a\u7814\u7a76\u4eba\u5458\u8ba4\u4e3a\u6062\u590d\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u662f\u6700\u7ec8\u76ee\u6807\uff0c\u56e0\u4e3a\u5b83\u6700\u63a5\u8fd1\u81ea\u7136\u751f\u7269\u5b66\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u672a\u6765\u7684\u7597\u6cd5\u80fd\u5426\u5b89\u5168\u5730\u5b9e\u73b0\u8fd9\u4e00\u76ee\u6807\uff0c\u76ee\u524d\u5c1a\u4e0d\u5f97\u800c\u77e5\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-families-continue-campaigning-for-elevidys\" class=\"wp-block-heading\">\u4e3a\u4ec0\u4e48\u5bb6\u5ead\u7ee7\u7eed\u4e3a Elevidys \u53d1\u8d77\u8fd0\u52a8<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u6279\u8bc4\u4eba\u58eb\u6709\u65f6\u4f1a\u8ba4\u4e3a\u5bb6\u5ead\u5bf9\u60c5\u51b5\u4e0d\u4e86\u89e3\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u79cd\u5047\u8bbe\u5f80\u5f80\u662f\u4e0d\u516c\u5e73\u7684\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5927\u591a\u6570\u5bb6\u5ead\u90fd\u660e\u767d\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u75c5\u60c5\u4f1a\u4e0d\u65ad\u6076\u5316\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6bcf\u4e00\u5e74\u90fd\u5f88\u91cd\u8981\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6bcf\u4e2a\u6708\u90fd\u5f88\u91cd\u8981\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7236\u6bcd\u5e38\u5e38\u9009\u62e9\u6cbb\u7597\uff0c\u56e0\u4e3a\u4ed6\u4eec\u5bb3\u6015\u5931\u53bb\u53ef\u80fd\u6c38\u8fdc\u4e0d\u4f1a\u518d\u56de\u6765\u7684\u673a\u4f1a\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u652f\u6301\u8005\u8ba4\u4e3a\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7b49\u5f85\u4f1a\u5e26\u6765\u98ce\u9669<\/li>\n\n\n\n<li>\u75be\u75c5\u8fdb\u5c55\u6301\u7eed<\/li>\n\n\n\n<li>\u672a\u6765\u7597\u6cd5\u5c1a\u4e0d\u660e\u6717\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u6000\u7591\u8bba\u8005\u8ba4\u4e3a\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u8bc1\u636e\u5c1a\u4e0d\u5b8c\u6574<\/li>\n\n\n\n<li>\u957f\u671f\u6548\u76ca\u5c1a\u4e0d\u660e\u786e\u3002<\/li>\n\n\n\n<li>\u672a\u6765\u7684\u7597\u6cd5\u6216\u8bb8\u80fd\u5e26\u6765\u66f4\u5927\u7684\u4f18\u52bf\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e24\u79cd\u89c2\u70b9\u90fd\u53ef\u4ee5\u7406\u89e3\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"questions-every-family-should-ask-before-choosing-elevidys\" class=\"wp-block-heading\">\u6bcf\u4e2a\u5bb6\u5ead\u5728\u9009\u62e9 Elevidys \u4e4b\u524d\u90fd\u5e94\u8be5\u95ee\u7684\u95ee\u9898<\/h2>\n\n\n\n<ol class=\"wp-block-list\">\n<li>\u6709\u54ea\u4e9b\u8bc1\u636e\u8bc1\u660e\u5176\u5177\u6709\u751f\u7269\u5b66\u76ca\u5904\uff1f<\/li>\n\n\n\n<li>\u6709\u54ea\u4e9b\u8bc1\u636e\u8bc1\u660e\u5176\u5177\u6709\u529f\u80fd\u6027\u76ca\u5904\uff1f<\/li>\n\n\n\n<li>\u6709\u54ea\u4e9b\u957f\u671f\u5b89\u5168\u6027\u6570\u636e\uff1f<\/li>\n\n\n\n<li>\u5982\u679c\u51fa\u73b0\u4e86\u66f4\u597d\u7684\u6cbb\u7597\u65b9\u6cd5\u4f1a\u600e\u6837\uff1f<\/li>\n\n\n\n<li>\u672a\u6765\u7684\u8d44\u683c\u4f1a\u53d7\u5230\u5f71\u54cd\u5417\uff1f<\/li>\n\n\n\n<li>\u6211\u7684\u5b69\u5b50\u76ee\u524d\u60a3\u6709\u54ea\u4e9b\u75be\u75c5\uff1f<\/li>\n\n\n\n<li>\u98ce\u9669\u6536\u76ca\u5206\u6790\u5177\u4f53\u5982\u4f55\u9002\u7528\u4e8e\u6211\u7684\u5b69\u5b50\uff1f<\/li>\n<\/ol>\n\n\n\n<p class=\"wp-block-paragraph\">\u4efb\u4f55\u793e\u4ea4\u5a92\u4f53\u89c6\u9891\u90fd\u65e0\u6cd5\u56de\u7b54\u8fd9\u4e9b\u95ee\u9898\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u53ea\u6709\u4ed4\u7ec6\u5ba1\u67e5\u8bc1\u636e\u5e76\u54a8\u8be2\u7ecf\u9a8c\u4e30\u5bcc\u7684\u795e\u7ecf\u808c\u8089\u4e13\u5bb6\u624d\u80fd\u505a\u5230\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\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5173\u4e8e Elevidys \u7684\u5e38\u89c1\u95ee\u9898\u89e3\u7b54<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"conclusion\" class=\"wp-block-heading\">\u7ed3\u8bba<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u56f4\u7ed5 Elevidys \u7684\u4e89\u8bba\u8bf4\u660e\u4e86\u73b0\u4ee3\u533b\u5b66\u9762\u4e34\u7684\u6700\u8270\u96be\u7684\u6311\u6218\u4e4b\u4e00\uff1a\u5728\u5145\u6ee1\u4e0d\u786e\u5b9a\u6027\u7684\u60c5\u51b5\u4e0b\u505a\u51fa\u6539\u53d8\u4eba\u751f\u7684\u51b3\u5b9a\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys \u80fd\u591f\u4ea7\u751f\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5e76\u5177\u6709\u751f\u7269\u6d3b\u6027\u8fd9\u4e00\u70b9\u6bcb\u5eb8\u7f6e\u7591\u3002\u957f\u671f\u968f\u8bbf\u6570\u636e\u4e5f\u8868\u660e\uff0c\u81f3\u5c11\u5728\u90e8\u5206\u60a3\u8005\u4e2d\uff0c\u75be\u75c5\u8fdb\u5c55\u53ef\u80fd\u5f97\u5230\u5ef6\u7f13\u3002\u7136\u800c\uff0c\u5173\u4e8e\u5176\u7597\u6548\u7684\u6301\u4e45\u6027\u3001\u5b89\u5168\u6027\u3001\u751f\u5b58\u83b7\u76ca\u3001\u6210\u672c\u6548\u76ca\u4ee5\u53ca\u5bf9\u672a\u6765\u6cbb\u7597\u9009\u62e9\u7684\u5f71\u54cd\u7b49\u91cd\u8981\u95ee\u9898\u4ecd\u7136\u5b58\u5728\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Elevidys\u7684\u652f\u6301\u8005\u7ecf\u5e38\u63f4\u5f15FDA\u7684\u83b7\u6279\u4ee5\u53ca\u5176\u4ee4\u4eba\u9f13\u821e\u7684\u4e34\u5e8a\u7ed3\u679c\u3002\u4e0e\u6b64\u540c\u65f6\uff0c\u6279\u8bc4\u8005\u5219\u8ba4\u4e3a\uff0c\u5728CK\u3001AST\u3001ALT\u7b49\u751f\u7269\u6807\u5fd7\u7269\u3001\u957f\u671f\u529f\u80fd\u9884\u540e\u4ee5\u53ca\u9884\u671f\u751f\u5b58\u671f\u7b49\u65b9\u9762\uff0c\u9700\u8981\u63d0\u9ad8\u900f\u660e\u5ea6\u3002\u8fd9\u4e9b\u60ac\u800c\u672a\u51b3\u7684\u95ee\u9898\u6301\u7eed\u5f15\u53d1\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7fa4\u4f53\u5185\u90e8\u7684\u4e89\u8bba\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Sarepta\u516c\u53f8\u80a1\u4ef7\u5728\u77ed\u77ed15\u4e2a\u6708\u5185\u4ece\u7ea6122\u7f8e\u5143\u66b4\u8dcc\u81f315\u7f8e\u5143\uff0c\u8fd9\u79cd\u5267\u70c8\u7684\u80a1\u4ef7\u6ce2\u52a8\u5bf9\u4e8e\u4e00\u5bb6\u751f\u7269\u6280\u672f\u516c\u53f8\u800c\u8a00\u5b9e\u5c5e\u7f55\u89c1\u3002\u867d\u7136\u80a1\u4ef7\u8868\u73b0\u672c\u8eab\u5e76\u4e0d\u80fd\u51b3\u5b9a\u4e00\u79cd\u7597\u6cd5\u662f\u5426\u6709\u6548\uff0c\u4f46\u5982\u6b64\u5de8\u5927\u7684\u5e02\u503c\u635f\u5931\u8868\u660e\uff0c\u8bb8\u591a\u6295\u8d44\u8005\u8d8a\u6765\u8d8a\u62c5\u5fe7\u73b0\u6709\u6570\u636e\u3001\u5b89\u5168\u6027\u8fdb\u5c55\u3001\u76d1\u7ba1\u4e0d\u786e\u5b9a\u6027\u3001\u672a\u6765\u7ade\u4e89\u4ee5\u53ca\u8be5\u7597\u6cd5\u7684\u6574\u4f53\u98ce\u9669\u6536\u76ca\u6bd4\u3002\u8fd9\u4e9b\u62c5\u5fe7\u6700\u7ec8\u662f\u5426\u5c5e\u5b9e\u8fd8\u6709\u5f85\u89c2\u5bdf\uff0c\u4f46\u80a1\u4ef7\u4e0b\u8dcc\u65e0\u7591\u53cd\u6620\u51fa\u6295\u8d44\u8005\u4fe1\u5fc3\u7684\u663e\u8457\u4e0b\u964d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\" id=\"conclusion\">\u7136\u800c\uff0c\u5bf9\u5bb6\u5ead\u800c\u8a00\uff0c\u6700\u91cd\u8981\u7684\u95ee\u9898\u5e76\u975e\u6295\u8d44\u8005\u662f\u5426\u4fe1\u4efbElevidys\uff0c\u800c\u662f\u73b0\u6709\u8bc1\u636e\u662f\u5426\u8db3\u4ee5\u652f\u6301\u7acb\u5373\u8fdb\u884c\u6cbb\u7597\uff0c\u6216\u8005\u7b49\u5f85\u66f4\u591a\u6570\u636e\u548c\u4e0b\u4e00\u4ee3\u7597\u6cd5\u662f\u5426\u624d\u662f\u66f4\u9002\u5408\u5b69\u5b50\u7684\u9009\u62e9\u3002\u6bcf\u4e2a\u5bb6\u5ead\u90fd\u5e94\u8be5\u5728\u505a\u51fa\u53ef\u80fd\u5f71\u54cd\u5b69\u5b50\u672a\u6765\u7684\u51b3\u5b9a\u4e4b\u524d\uff0c\u83b7\u5f97\u900f\u660e\u7684\u4fe1\u606f\u3001\u5766\u8bda\u7684\u8ba8\u8bba\u4ee5\u53ca\u6240\u6709\u53ef\u7528\u7684\u8bc1\u636e\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6700\u540e\u4e00\u70b9\uff1a\u5982\u679c\u6709\u4eba\u544a\u8bc9\u4f60\u201cCK \u4e0d\u91cd\u8981\u201d\uff0c\u4e0d\u8981\u5728\u6ca1\u6709\u8bc1\u636e\u7684\u60c5\u51b5\u4e0b\u8f7b\u6613\u63a5\u53d7\u8fd9\u79cd\u8bf4\u6cd5\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u662f\u4e00\u79cd\u4ee5\u6301\u7eed\u6027\u808c\u8089\u635f\u4f24\u4e3a\u7279\u5f81\u7684\u75be\u75c5\uff0c\u800c CK \u6b63\u662f\u8fd9\u79cd\u635f\u4f24\u6700\u660e\u663e\u7684\u6307\u6807\u4e4b\u4e00\u3002.<\/strong> \u5982\u679c\u808c\u9178\u6fc0\u9176 (CK) \u6c34\u5e73\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8bca\u65ad\u548c\u76d1\u6d4b\u4e2d\u81f3\u5173\u91cd\u8981\uff0c\u90a3\u4e48\u5728 Elevidys \u624b\u672f\u540e\uff0c\u5176\u91cd\u8981\u6027\u4f9d\u7136\u4e0d\u51cf\u3002CK \u6c34\u5e73\u53d8\u5316\u8d8b\u52bf\u7684\u900f\u660e\u5ea6\u5e94\u88ab\u89c6\u4e3a\u4e00\u9879\u5fc5\u8981\u63aa\u65bd\uff0c\u800c\u975e\u53ef\u9009\u9879\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4f5c\u4e3a DMDWarrior\uff0c\u6211\u4eec\u5411 Sarepta \u63d0\u4ea4\u4e86\u4e00\u7cfb\u5217\u95ee\u9898\uff0c\u8fd9\u4e9b\u95ee\u9898\u6d89\u53ca\u8bb8\u591a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5bb6\u5ead\u7ecf\u5e38\u8be2\u95ee\u7684\u4e3b\u9898\uff0c\u5305\u62ec\u4e34\u5e8a\u7ed3\u679c\u3001\u751f\u7269\u6807\u5fd7\u7269\u3001\u957f\u671f\u9884\u671f\u548c\u672a\u6765\u6cbb\u7597\u8003\u8651\u56e0\u7d20\u3002.<\/strong> \u6211\u4eec\u88ab\u544a\u77e5\u5c06\u4f1a\u4f5c\u51fa\u56de\u5e94\uff0c\u4f46\u622a\u81f3\u672c\u6587\u53d1\u8868\u65f6\uff0c\u6211\u4eec\u5c1a\u672a\u6536\u5230\u8fd9\u4e9b\u95ee\u9898\u7684\u8be6\u7ec6\u7b54\u590d\u3002\u5982\u679cSarepta\u516c\u53f8\u9009\u62e9\u5728\u4efb\u4f55\u65f6\u5019\u4f5c\u51fa\u56de\u5e94\uff0c\u6211\u4eec\u51c6\u5907\u5168\u6587\u520a\u767b\u5176\u7b54\u590d\uff0c\u4ee5\u4fbf\u60a3\u8005\u3001\u5bb6\u5c5e\u548c\u533b\u7597\u4e13\u4e1a\u4eba\u5458\u80fd\u591f\u76f4\u63a5\u67e5\u9605\u76f8\u5173\u4fe1\u606f\u5e76\u5f97\u51fa\u81ea\u5df1\u7684\u7ed3\u8bba\u3002.<\/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>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>Few Duchenne muscular dystrophy (DMD) treatments have generated as much excitement, controversy, and debate as Elevidys (delandistrogene moxeparvovec), the gene therapy developed by Sarepta Therapeutics. For many families, Elevidys represents hope. It is the first approved gene therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy by delivering a micro-dystrophin gene to [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8120,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[33,743,179,742,821],"class_list":["post-8116","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-elevidys","tag-elevidys-cost","tag-elevidys-gene-therapy","tag-elevidys-price","tag-sarepta-stock"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8116","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=8116"}],"version-history":[{"count":9,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8116\/revisions"}],"predecessor-version":[{"id":8126,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8116\/revisions\/8126"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/8120"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=8116"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=8116"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=8116"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}