{"id":7875,"date":"2026-05-08T10:37:41","date_gmt":"2026-05-08T07:37:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7875"},"modified":"2026-05-08T10:37:43","modified_gmt":"2026-05-08T07:37:43","slug":"bmn-351-exon-51-skipping-therapy-phase-2-clinical-trial-nct07573631","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/bmn-351-exon-51-skipping-therapy-phase-2-clinical-trial-nct07573631\/","title":{"rendered":"BioMarin\u516c\u53f8\u542f\u52a8BMN 351\u5916\u663e\u5b5051\u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e8c\u671f\u4e34\u5e8a\u8bd5\u9a8c"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>BioMarin Pharmaceutical \u5df2\u6b63\u5f0f\u542f\u52a8\u4e00\u9879\u65b0\u7684 2 \u671f\u6269\u5c55\u7814\u7a76 (NCT07573631)\uff0c\u8bc4\u4f30 BMN 351\uff0c\u8fd9\u662f\u4e00\u79cd\u7814\u7a76\u6027\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\uff0c\u65e8\u5728\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u3002.<\/strong> \u8be5\u7814\u7a76\u5c06\u91cd\u70b9\u5173\u6ce8\u5148\u524d\u5b8c\u6210 351-201 \u4e34\u5e8a\u8bd5\u9a8c\u7684\u53c2\u4e0e\u8005\u7684\u957f\u671f\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u8eab\u4f53\u529f\u80fd\u7ed3\u679c\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">BMN 351 Exon 51 \u8df3\u8dc3\u7597\u6cd5 2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>BMN 351 \u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5 2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u662f\u4e00\u9879\u591a\u4e2d\u5fc3\u3001\u5f00\u653e\u6807\u7b7e\u7684\u6269\u5c55\u7814\u7a76\uff0c\u9488\u5bf9\u9002\u5408\u63a5\u53d7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u3002.<\/strong> \u7814\u7a76\u4eba\u5458\u5c06\u8bc4\u4f30\u6bcf\u5468\u4e00\u6b21\u7684\u9759\u8109\u6ce8\u5c04 BMN 351 \u5728\u8f83\u957f\u7684\u6cbb\u7597\u671f\u5185\u662f\u5426\u4ecd\u7136\u5b89\u5168\u6709\u6548\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u8bd5\u9a8c\u8ba1\u5212\u62db\u52df\u6700\u591a 18 \u540d\u5e74\u9f84\u5728 4 \u5c81\u81f3 23 \u5c81\u4e4b\u95f4\u7684\u53c2\u4e0e\u8005\uff0c\u4ed6\u4eec\u4e4b\u524d\u5df2\u5b8c\u6210\u540d\u4e3a 351-201 \u7684 1\/2 \u671f\u7814\u7a76\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u4e86\u89e3\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u548c\u5916\u663e\u5b5051\u8df3\u8dc3<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">\u4ec0\u4e48\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff1f<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u662f\u4e00\u79cd\u7f55\u89c1\u7684\u9057\u4f20\u6027\u75be\u75c5\uff0c\u4f1a\u5bfc\u81f4\u8fdb\u884c\u6027\u808c\u8089\u65e0\u529b\u548c\u808c\u8089\u9000\u5316\u3002\u5b83\u662f\u7531\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7a81\u53d8\u5f15\u8d77\u7684\uff0c\u800c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u5bf9\u4e8e\u7ef4\u6301\u5065\u5eb7\u7684\u808c\u8089\u7ec6\u80de\u81f3\u5173\u91cd\u8981\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u4ec0\u4e48\u662f\u5916\u663e\u5b5051\u8df3\u8dc3\uff1f<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u65e8\u5728\u5e2e\u52a9\u4eba\u4f53\u4ea7\u751f\u4e00\u79cd\u8f83\u77ed\u4f46\u529f\u80fd\u6b63\u5e38\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u5916\u663e\u5b5051\u8df3\u8dc3\u7597\u6cd5\u4e13\u95e8\u9488\u5bf9\u90a3\u4e9b\u57fa\u56e0\u7a81\u53d8\u53ef\u80fd\u53d7\u76ca\u4e8e\u5728\u86cb\u767d\u8d28\u5408\u6210\u8fc7\u7a0b\u4e2d\u8df3\u8fc7\u5916\u663e\u5b5051\u7684\u60a3\u8005\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">BMN 351 \u6b63\u5728\u88ab\u7814\u7a76\u4f5c\u4e3a\u4e00\u79cd\u65b0\u4e00\u4ee3\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\uff0c\u53ef\u80fd\u6709\u52a9\u4e8e\u51cf\u7f13\u7b26\u5408\u6761\u4ef6\u7684\u60a3\u8005\u7684\u75be\u75c5\u8fdb\u5c55\u3002.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"847\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg\" alt=\"\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u53ef\u901a\u8fc7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u7a81\u53d8\u548c\u7f3a\u5931\" class=\"wp-image-4526\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg 847w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-248x300.jpg 248w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-768x929.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1270x1536.jpg 1270w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1693x2048.jpg 1693w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-10x12.jpg 10w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-347x420.jpg 347w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-150x181.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-300x363.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-696x842.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1068x1292.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1920x2323.jpg 1920w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping.jpg 1984w\" sizes=\"auto, (max-width: 847px) 100vw, 847px\" \/><\/figure>\n\n\n\n<h2 class=\"wp-block-heading\">\u4e8c\u671f\u7814\u7a76\u7684\u5173\u952e\u7ec6\u8282<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">\u7814\u7a76\u8bbe\u8ba1<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5ba1\u5224\u5185\u5bb9\u5982\u4e0b\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u591a\u4e2d\u5fc3<\/li>\n\n\n\n<li>\u5f00\u653e\u6807\u7b7e<\/li>\n\n\n\n<li>\u957f\u671f\u6269\u5c55\u7814\u7a76<\/li>\n\n\n\n<li>\u4e13\u6ce8\u4e8e\u6bcf\u5468\u4e00\u6b21\u7684\u9759\u8109\u6ce8\u5c04\u7ed9\u836f<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u672c\u7814\u7a76\u7684\u7b2c\u4e00\u6b21\u8bbf\u89c6\u4e5f\u5c06\u4f5c\u4e3a\u65e9\u671f 351-201 \u8bd5\u9a8c\u7684\u6700\u540e\u4e00\u6b21\u8bbf\u89c6\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u9884\u8ba1\u65f6\u95f4\u8868<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5f00\u8bfe\u65f6\u95f4\uff1a2026\u5e744\u6708<\/li>\n\n\n\n<li>\u4e3b\u8981\u7ae3\u5de5\u65e5\u671f\uff1a2031\u5e7412\u6708<\/li>\n\n\n\n<li>\u9884\u8ba1\u7814\u7a76\u5b8c\u6210\u65f6\u95f4\uff1a2031\u5e7412\u6708<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">\u53c2\u4e0e\u8fd9\u9879\u7814\u7a76\u7684\u56fd\u5bb6<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7814\u7a76\u5305\u62ec\u4ee5\u4e0b\u4e34\u5e8a\u8bd5\u9a8c\u5730\u70b9\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u610f\u5927\u5229<\/li>\n\n\n\n<li>\u8377\u5170<\/li>\n\n\n\n<li>\u897f\u73ed\u7259<\/li>\n\n\n\n<li>\u571f\u8033\u5176<\/li>\n\n\n\n<li>\u82f1\u56fd<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">\u8c01\u53ef\u4ee5\u53c2\u52a0\uff1f<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\">\u4e3b\u8981\u7eb3\u5165\u6807\u51c6<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u53c2\u4e0e\u8005\u5fc5\u987b\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5df2\u5b8c\u6210 351-201 \u7814\u7a76<\/li>\n\n\n\n<li>\u7ee7\u7eed\u7a33\u5b9a\u7684\u76ae\u8d28\u7c7b\u56fa\u9187\u6cbb\u7597<\/li>\n\n\n\n<li>\u9075\u5faa\u8003\u5bdf\u8bbf\u95ee\u8981\u6c42<\/li>\n\n\n\n<li>\u5728\u9002\u7528\u60c5\u51b5\u4e0b\uff0c\u63d0\u4f9b\u77e5\u60c5\u540c\u610f\u6216\u8ba4\u53ef\u3002<\/li>\n\n\n\n<li>\u5982\u679c\u65b9\u6848\u6307\u5357\u8981\u6c42\uff0c\u8bf7\u4f7f\u7528\u907f\u5b55\u63aa\u65bd<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\">\u4e3b\u8981\u6392\u9664\u6807\u51c6<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u7b26\u5408\u4ee5\u4e0b\u6761\u4ef6\u8005\u4e0d\u5f97\u53c2\u52a0\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5df2\u77e5\u6709\u51dd\u8840\u969c\u788d<\/li>\n\n\n\n<li>\u4f7f\u7528\u8fdd\u7981\u836f\u7269<\/li>\n\n\n\n<li>\u5728\u57fa\u7ebf\u68c0\u67e5\u524d 12 \u5468\u5185\u63a5\u53d7\u4e86\u53e6\u4e00\u9879\u5df2\u83b7\u6279\u51c6\u7684\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u3002<\/li>\n\n\n\n<li>\u66fe\u63a5\u53d7\u8fc7\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u57fa\u56e0\u6cbb\u7597\u3002<\/li>\n\n\n\n<li>\u4f7f\u7528\u6297\u51dd\u8840\u5242\u6216\u514d\u75ab\u6291\u5236\u5242<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">\u8fd9\u573a\u5ba1\u5224\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7fa4\u4f53\u4e3a\u4f55\u5982\u6b64\u91cd\u8981<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>BMN 351 \u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5 2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u7684\u542f\u52a8\uff0c\u6807\u5fd7\u7740\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u9776\u5411\u57fa\u56e0\u7597\u6cd5\u53d1\u5c55\u53c8\u8fc8\u51fa\u4e86\u91cd\u8981\u4e00\u6b65\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u957f\u671f\u6269\u5c55\u7814\u7a76\u5c24\u4e3a\u91cd\u8981\uff0c\u56e0\u4e3a\u5b83\u4eec\u6709\u52a9\u4e8e\u7814\u7a76\u4eba\u5458\u4e86\u89e3\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6cbb\u7597\u6548\u679c\u80fd\u5426\u957f\u671f\u7ef4\u6301<\/li>\n\n\n\n<li>\u957f\u671f\u5b89\u5168\u6027\u6982\u51b5<\/li>\n\n\n\n<li>\u5bf9\u65e5\u5e38\u8eab\u4f53\u673a\u80fd\u7684\u5f71\u54cd<\/li>\n\n\n\n<li>\u5bf9\u75be\u75c5\u8fdb\u5c55\u7684\u6f5c\u5728\u5f71\u54cd<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bf9\u4e8e\u53d7\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5f71\u54cd\u7684\u5bb6\u5ead\u6765\u8bf4\uff0c\u5916\u663e\u5b50\u8df3\u8dc3\u7814\u7a76\u7684\u4e0d\u65ad\u8fdb\u5c55\u4e3a\u672a\u6765\u7684\u6cbb\u7597\u9009\u62e9\u5e26\u6765\u4e86\u65b0\u7684\u5e0c\u671b\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>","protected":false},"excerpt":{"rendered":"<p>BioMarin Pharmaceutical has officially launched a new Phase 2 extension study (NCT07573631) evaluating BMN 351, an investigational exon 51 skipping therapy designed for people living with Duchenne muscular dystrophy (DMD). The study will focus on long-term safety, tolerability, and physical function outcomes in participants who previously completed the earlier 351-201 clinical trial. What Is the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7877,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[759,760,137,122,193,421,194],"class_list":["post-7875","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-biomarin","tag-bmn-351","tag-clinical-trial","tag-exon-51","tag-exon-51-skipping","tag-exon-51-skipping-therapy","tag-exon-51-treatment"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/7875","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=7875"}],"version-history":[{"count":2,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/7875\/revisions"}],"predecessor-version":[{"id":7878,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/7875\/revisions\/7878"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/7877"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=7875"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=7875"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=7875"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}