{"id":8259,"date":"2026-07-20T15:10:42","date_gmt":"2026-07-20T12:10:42","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=8259"},"modified":"2026-07-20T15:30:25","modified_gmt":"2026-07-20T12:30:25","slug":"z-rostudirsen-fda-bla-accepted","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/z-rostudirsen-fda-bla-accepted\/","title":{"rendered":"Dyne Therapeutics \u5ba3\u5e03\u7f8e\u56fd FDA \u53d7\u7406 Z-Rostudirsen \u7684\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7 (BLA)\uff0cZ-Rostudirsen \u662f\u4e00\u79cd\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u3002"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>\u53d7\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u5f71\u54cd\u7684\u5bb6\u5ead\u6536\u5230\u4e86\u4ee4\u4eba\u9f13\u821e\u7684\u6d88\u606f\uff0cDyne Therapeutics \u5ba3\u5e03\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u5df2\u63a5\u53d7\u5176 z-rostudirsen\uff08\u4ee5\u524d\u79f0\u4e3a DYNE-251\uff09\u7684\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7 (BLA)\u3002.<\/strong> \u8be5\u8bd5\u9a8c\u6027\u7597\u6cd5\u6b63\u5728\u4e3a\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u624b\u672f\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u5f00\u53d1\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u8fd8\u6388\u4e88\u4e86\u4f18\u5148\u5ba1\u8bc4\u8d44\u683c\uff0c\u7f29\u77ed\u4e86\u5ba1\u8bc4\u65f6\u95f4\uff0c\u5e76\u5c06\u5904\u65b9\u836f\u7528\u6237\u6536\u8d39\u6cd5\u6848 (PDUFA) \u7684\u76ee\u6807\u884c\u52a8\u65e5\u671f\u8bbe\u5b9a\u4e3a 2027 \u5e74 1 \u6708 21 \u65e5\u3002\u5982\u679c\u83b7\u5f97\u6279\u51c6\uff0cDyne \u9884\u8ba1\u5c06\u4e8e 2027 \u5e74\u7b2c\u4e00\u5b63\u5ea6\u5728\u7f8e\u56fd\u63a8\u51fa z-rostudirsen\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=\"#what-is-z-rostudirsen\">\u4ec0\u4e48\u662f Z-Rostudirsen\uff1f<\/a><\/li><li><a href=\"#fda-accepts-biologics-license-application\">FDA \u63a5\u53d7\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7<\/a><\/li><li><a href=\"#why-is-accelerated-approval-important\">\u4e3a\u4ec0\u4e48\u52a0\u901f\u5ba1\u6279\u5982\u6b64\u91cd\u8981\uff1f<\/a><\/li><li><a href=\"#deliver-trial-results\">DELIVER\u8bd5\u9a8c\u7ed3\u679c<\/a><\/li><li><a href=\"#dyne-therapeutics-plans-u-s-launch-in-early-2027\">Dyne Therapeutics\u8ba1\u5212\u4e8e2027\u5e74\u521d\u5728\u7f8e\u56fd\u4e0a\u5e02\u3002<\/a><\/li><li><a href=\"#dynes-broader-duchenne-pipeline\">\u6234\u6069\u516c\u53f8\u7684\u5927\u675c\u5174\u6cb3\u7ba1\u9053<\/a><\/li><li><a href=\"#what-this-means-for-the-duchenne-community\">\u8fd9\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7fa4\u4f53\u610f\u5473\u7740\u4ec0\u4e48<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"what-is-z-rostudirsen\" class=\"wp-block-heading\">\u4ec0\u4e48\u662f Z-Rostudirsen\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Z-rostudirsen\uff08\u4ee5\u524d\u79f0\u4e3a DYNE-251\uff09\u662f\u4e00\u79cd\u7814\u7a76\u6027\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\uff0c\u65e8\u5728\u6cbb\u7597\u7531\u9002\u5408\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684\u7a81\u53d8\u5f15\u8d77\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7597\u6cd5\u91c7\u7528Dyne Therapeutics\u516c\u53f8\u4e13\u6709\u7684FORCE\u2122\u5e73\u53f0\uff0c\u65e8\u5728\u63d0\u9ad8\u6cbb\u7597\u5206\u5b50\u76f4\u63a5\u8f93\u9001\u81f3\u9aa8\u9abc\u808c\u548c\u5fc3\u808c\u7ec6\u80de\u7684\u6548\u7387\u3002\u901a\u8fc7\u589e\u5f3a\u836f\u7269\u5411\u808c\u8089\u7ec4\u7ec7\u7684\u8f93\u9001\uff0c\u8be5\u7597\u6cd5\u65e8\u5728\u589e\u52a0\u8fd1\u4e4e\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u751f\u6210\uff0c\u800c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5bf9\u4e8e\u7ef4\u6301\u5065\u5eb7\u7684\u808c\u8089\u529f\u80fd\u81f3\u5173\u91cd\u8981\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0e\u53ef\u80fd\u9650\u5236\u808c\u8089\u5438\u6536\u7684\u4f20\u7edf\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u4e0d\u540c\uff0cz-rostudirsen \u7ecf\u8fc7\u4e13\u95e8\u8bbe\u8ba1\uff0c\u53ef\u5728\u4fdd\u6301\u826f\u597d\u5b89\u5168\u6027\u7684\u540c\u65f6\u6539\u5584\u7ec4\u7ec7\u8f93\u9001\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/mutations-and-deletions-amenable-to-exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u9002\u5408\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u7684\u7a81\u53d8\u548c\u7f3a\u5931<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"fda-accepts-biologics-license-application\" class=\"wp-block-heading\">FDA \u63a5\u53d7\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u53d7\u7406\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7 (BLA) \u610f\u5473\u7740\u8be5\u673a\u6784\u5df2\u8ba4\u5b9a\u8be5\u7533\u8bf7\u6750\u6599\u9f50\u5168\uff0c\u53ef\u4ee5\u5f00\u59cb\u6b63\u5f0f\u7684\u79d1\u5b66\u5ba1\u67e5\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/biologics-license-application-bla\/\" target=\"_blank\" data-type=\"post\" data-id=\"5027\" rel=\"noreferrer noopener\">\u751f\u7269\u5236\u54c1\u8bb8\u53ef\u7533\u8bf7\uff08BLA\uff09<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4f5c\u4e3a\u6b64\u8fc7\u7a0b\u7684\u4e00\u90e8\u5206\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>FDA \u6388\u4e88\u4f18\u5148\u5ba1\u67e5\u6743\uff0c\u7f29\u77ed\u4e86\u6807\u51c6\u5ba1\u67e5\u671f\u3002.<\/strong><\/li>\n\n\n\n<li>PDUFA \u76ee\u6807\u751f\u6548\u65e5\u671f\u5df2\u5b9a\u4e3a 2027 \u5e74 1 \u6708 21 \u65e5\u3002.<\/li>\n\n\n\n<li><strong>\u8be5\u7533\u8bf7\u5bfb\u6c42\u4ee5\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u4e3a\u66ff\u4ee3\u7ec8\u70b9\u7684\u52a0\u901f\u5ba1\u6279\u3002.<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c FDA \u7814\u7a76\u5f97\u51fa\u7ed3\u8bba\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u589e\u52a0\u5f88\u53ef\u80fd\u9884\u6d4b\u4e34\u5e8a\u83b7\u76ca\uff0c\u90a3\u4e48 z-rostudirsen \u53ef\u80fd\u4f1a\u83b7\u5f97\u52a0\u901f\u6279\u51c6\uff0c\u540c\u65f6\u5176\u4ed6\u7814\u7a76\u5c06\u7ee7\u7eed\u8bc1\u5b9e\u957f\u671f\u4e34\u5e8a\u7ed3\u679c\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-is-accelerated-approval-important\" class=\"wp-block-heading\">\u4e3a\u4ec0\u4e48\u52a0\u901f\u5ba1\u6279\u5982\u6b64\u91cd\u8981\uff1f<\/h2>\n\n\n\n<h3 id=\"understanding-the-accelerated-approval-pathway\" class=\"wp-block-heading\">\u4e86\u89e3\u52a0\u901f\u5ba1\u6279\u9014\u5f84<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f00\u53d1\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7b49\u7f55\u89c1\u75be\u75c5\u7684\u65b9\u6cd5\u901a\u5e38\u9700\u8981\u5f88\u591a\u5e74\u65f6\u95f4\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3a\u4e86\u5e2e\u52a9\u60a3\u8005\u5c3d\u65e9\u83b7\u5f97\u6709\u524d\u666f\u7684\u7597\u6cd5\uff0cFDA \u521b\u5efa\u4e86\u52a0\u901f\u5ba1\u6279\u9014\u5f84\u3002\u8be5\u76d1\u7ba1\u9014\u5f84\u5141\u8bb8\u57fa\u4e8e\u80fd\u591f\u5408\u7406\u9884\u6d4b\u4e34\u5e8a\u83b7\u76ca\u7684\u66ff\u4ee3\u7ec8\u70b9\u8fdb\u884c\u5ba1\u6279\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bf9\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u5df2\u6210\u4e3a\u76d1\u7ba1\u673a\u6784\u8bc4\u4f30\u7684\u6700\u91cd\u8981\u7684\u66ff\u4ee3\u751f\u7269\u6807\u5fd7\u7269\u4e4b\u4e00\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u83b7\u5f97\u52a0\u901f\u6279\u51c6\u7684\u516c\u53f8\u901a\u5e38\u9700\u8981\u7ee7\u7eed\u8fdb\u884c\u4e0a\u5e02\u540e\u7814\u7a76\uff0c\u4ee5\u786e\u8ba4\u60a3\u8005\u80fd\u591f\u83b7\u5f97\u6709\u610f\u4e49\u7684\u957f\u671f\u4e34\u5e8a\u76ca\u5904\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7acb\u5373\u4f7f\u7528\u6211\u4eec\u7684\u5de5\u5177<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/duchenne-exon-deletion-tool\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD\/BMD \u5916\u663e\u5b50\u5206\u6790\u5de5\u5177<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"deliver-trial-results\" class=\"wp-block-heading\">DELIVER\u8bd5\u9a8c\u7ed3\u679c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">BLA \u7533\u8bf7\u5f97\u5230\u4e86 1\/2 \u671f DELIVER \u4e34\u5e8a\u8bd5\u9a8c\u6ce8\u518c\u6269\u5c55\u961f\u5217\u7ed3\u679c\u7684\u652f\u6301\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6839\u636e Dyne Therapeutics \u7814\u7a76\uff0c\u6bcf\u56db\u5468\u63a5\u53d7\u4e00\u6b21 z-rostudirsen \u6cbb\u7597\u7684\u53c2\u4e0e\u8005\u8868\u73b0\u51fa\uff1a<\/p>\n\n\n\n<h3 id=\"significant-increase-in-dystrophin\" class=\"wp-block-heading\">\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u663e\u8457\u589e\u52a0<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u60a3\u8005\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ea7\u91cf\u51fa\u73b0\u4e86\u663e\u8457\u7684\u7edf\u8ba1\u5b66\u610f\u4e49\u4e0a\u7684\u589e\u957f\uff0c\u8fd9\u652f\u6301\u4e86\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u7684\u4e3b\u8981\u751f\u7269\u5b66\u76ee\u6807\u3002.<\/p>\n\n\n\n<h3 id=\"improvements-across-multiple-functional-measures\" class=\"wp-block-heading\">\u591a\u9879\u529f\u80fd\u6307\u6807\u5747\u6709\u6240\u6539\u5584<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u9664\u4e86\u5b9e\u9a8c\u5ba4\u7814\u7a76\u7ed3\u679c\u5916\uff0c\u7814\u7a76\u4eba\u5458\u8fd8\u89c2\u5bdf\u5230\u591a\u4e2a\u4e34\u5e8a\u529f\u80fd\u7ec8\u70b9\u6307\u6807\u6709\u6240\u6539\u5584\uff0c\u8fd9\u8868\u660e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u589e\u52a0\u53ef\u80fd\u8f6c\u5316\u4e3a\u66f4\u597d\u7684\u8eab\u4f53\u673a\u80fd\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c3d\u7ba1\u5176\u4ed6\u957f\u671f\u7814\u7a76\u4ecd\u5728\u8fdb\u884c\u4e2d\uff0c\u4f46\u8fd9\u4e9b\u529f\u80fd\u6027\u53d1\u73b0\u52a0\u5f3a\u4e86\u652f\u6301\u8be5\u7597\u6cd5\u7684\u603b\u4f53\u8bc1\u636e\u3002.<\/p>\n\n\n\n<h3 id=\"favorable-safety-profile\" class=\"wp-block-heading\">\u826f\u597d\u7684\u5b89\u5168\u6027<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u8fd8\u62a5\u544a\u79f0\uff0c\u8be5\u836f\u7269\u5177\u6709\u826f\u597d\u7684\u5b89\u5168\u6027\uff0c\u5728\u6574\u4e2a\u7814\u7a76\u671f\u95f4\uff0c\u60a3\u8005\u5bf9\u6cbb\u7597\u7684\u8010\u53d7\u6027\u666e\u904d\u826f\u597d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728 FDA \u5ba1\u67e5\u8fc7\u7a0b\u4e2d\uff0c\u5b89\u5168\u6027\u4ecd\u7136\u662f\u5173\u952e\u8003\u8651\u56e0\u7d20\u4e4b\u4e00\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"dyne-therapeutics-plans-u-s-launch-in-early-2027\" class=\"wp-block-heading\">Dyne Therapeutics\u8ba1\u5212\u4e8e2027\u5e74\u521d\u5728\u7f8e\u56fd\u4e0a\u5e02\u3002<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5047\u8bbe FDA \u7684\u6279\u51c6\u6309\u8ba1\u5212\u83b7\u5f97\u6279\u51c6\uff0cDyne Therapeutics \u9884\u8ba1\u5c06\u4e8e 2027 \u5e74\u7b2c\u4e00\u5b63\u5ea6\u5728\u7f8e\u56fd\u63a8\u51fa z-rostudirsen\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u516c\u53f8\u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98\u7ea6\u7ff0\u00b7\u8003\u514b\u65af\u8868\u793a\uff0c\u76ee\u6807\u662f\u63d0\u4f9b\u4e00\u79cd\u80fd\u591f\u5b9e\u73b0\u4ee5\u4e0b\u76ee\u6807\u7684\u7597\u6cd5\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5927\u91cf\u4ea7\u751f\u63a5\u8fd1\u5168\u957f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d<\/li>\n\n\n\n<li>\u5e7f\u6cdb\u8f93\u9001\u81f3\u76f8\u5173\u808c\u8089\u7ec4\u7ec7<\/li>\n\n\n\n<li>\u6539\u5584\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7684\u529f\u80fd<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u8fd8\u80af\u5b9a\u4e86\u60a3\u8005\u3001\u5bb6\u5c5e\u3001\u7814\u7a76\u4eba\u5458\u3001\u5021\u5bfc\u7ec4\u7ec7\u4ee5\u53ca\u66f4\u5e7f\u6cdb\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7fa4\u4f53\u5728\u6574\u4e2a\u4e34\u5e8a\u5f00\u53d1\u8fc7\u7a0b\u4e2d\u53d1\u6325\u7684\u91cd\u8981\u4f5c\u7528\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"dynes-broader-duchenne-pipeline\" class=\"wp-block-heading\">\u6234\u6069\u516c\u53f8\u7684\u5927\u675c\u5174\u6cb3\u7ba1\u9053<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Z-rostudirsen \u53ea\u662f Dyne Therapeutics&#039; \u66f4\u5e7f\u6cdb\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5f00\u53d1\u8ba1\u5212\u7684\u4e00\u90e8\u5206\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u516c\u53f8\u8fd8\u5728\u63a8\u8fdb\u5176\u4ed6\u51e0\u79cd\u9488\u5bf9\u5176\u4ed6\u57fa\u56e0\u7a81\u53d8\u7684\u5728\u7814\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u3002.<\/p>\n\n\n\n<h3 id=\"dyne-253\" class=\"wp-block-heading\">DYNE-253<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e13\u4e3a\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 53 \u8df3\u8dc3\u624b\u672f\u7684\u60a3\u8005\u8bbe\u8ba1\u3002.<\/p>\n\n\n\n<h3 id=\"dyne-245\" class=\"wp-block-heading\">DYNE-245<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e13\u4e3a\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 45 \u8df3\u8dc3\u624b\u672f\u7684\u60a3\u8005\u8bbe\u8ba1\u3002.<\/p>\n\n\n\n<h3 id=\"dyne-244\" class=\"wp-block-heading\">DYNE-244<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e13\u4e3a\u9002\u5408\u8fdb\u884c\u7b2c 44 \u5916\u663e\u5b50\u8df3\u8dc3\u624b\u672f\u7684\u60a3\u8005\u8bbe\u8ba1\u3002.<\/p>\n\n\n\n<h3 id=\"dyne-255\" class=\"wp-block-heading\">DYNE-255<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e13\u4e3a\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 55 \u8df3\u8dc3\u624b\u672f\u7684\u60a3\u8005\u8bbe\u8ba1\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c\u6210\u529f\uff0c\u8fd9\u4e9b\u9879\u76ee\u53ef\u4ee5\u4e3a\u66f4\u591a\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4eba\u4eec\u63d0\u4f9b\u6cbb\u7597\u9009\u62e9\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"what-this-means-for-the-duchenne-community\" class=\"wp-block-heading\">\u8fd9\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7fa4\u4f53\u610f\u5473\u7740\u4ec0\u4e48<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">FDA \u83b7\u5f97 BLA \u6279\u51c6\u4ee3\u8868\u7740\u4e00\u4e2a\u91cd\u8981\u7684\u76d1\u7ba1\u91cc\u7a0b\u7891\uff0c\u800c\u4e0d\u662f\u6700\u7ec8\u6279\u51c6\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u7136\u800c\uff0c\u8fd9\u8868\u660e\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>FDA \u5df2\u5f00\u59cb\u5bf9 z-rostudirsen \u8fdb\u884c\u6b63\u5f0f\u5ba1\u67e5\u3002.<\/strong><\/li>\n\n\n\n<li>\u4f18\u5148\u5ba1\u67e5\u53cd\u6620\u4e86\u8be5\u7597\u6cd5\u7684\u6f5c\u5728\u91cd\u8981\u6027\u3002.<\/li>\n\n\n\n<li>\u8be5\u7533\u8bf7\u5f97\u5230\u4e86\u5177\u6709\u7edf\u8ba1\u5b66\u610f\u4e49\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u751f\u6210\u6570\u636e\u548c\u4ee4\u4eba\u9f13\u821e\u7684\u529f\u80fd\u6027\u7ed3\u679c\u7684\u652f\u6301\u3002.<\/li>\n\n\n\n<li><strong>\u9884\u8ba1\u5c06\u4e8e 2027 \u5e74 1 \u6708 21 \u65e5\u505a\u51fa\u76d1\u7ba1\u51b3\u5b9a\u3002.<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5982\u679c\u83b7\u5f97\u6279\u51c6\uff0cz-rostudirsen \u53ef\u80fd\u6210\u4e3a\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7684\u53e6\u4e00\u79cd\u6cbb\u7597\u9009\u62e9\uff0c\u8fdb\u4e00\u6b65\u6269\u5927\u8fd9\u79cd\u6bc1\u706d\u6027\u9057\u4f20\u75be\u75c5\u7684\u6cbb\u7597\u8303\u56f4\u3002.<\/strong><\/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\/clinical-trials\/\" 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>Families affected by Duchenne muscular dystrophy (DMD) received encouraging news as Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for z-rostudirsen (formerly DYNE-251). The investigational therapy is being developed for people with Duchenne muscular dystrophy amenable to exon 51 skipping. The FDA has also granted [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":8260,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,122,193,421,194,460],"class_list":["post-8259","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment","tag-z-rostudirsen"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8259","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=8259"}],"version-history":[{"count":4,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8259\/revisions"}],"predecessor-version":[{"id":8266,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/8259\/revisions\/8266"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/8260"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=8259"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=8259"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=8259"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}