{"id":4274,"date":"2025-08-04T17:03:02","date_gmt":"2025-08-04T14:03:02","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4274"},"modified":"2025-08-04T17:13:24","modified_gmt":"2025-08-04T14:13:24","slug":"dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd\/","title":{"rendered":"Dyne Therapeutics \u5ba3\u5e03 FDA \u83b7\u5f97 DYNE-251 \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics\u516c\u53f8\u5ba3\u5e03\uff0c\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u5df2\u6388\u4e88DYNE-251\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u53ef\u8fdb\u884c51\u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u3002\u8be5\u8ba4\u5b9a\u57fa\u4e8e\u6b63\u5728\u8fdb\u884c\u7684DELIVER\u4e34\u5e8a\u8bd5\u9a8c\u7684\u6570\u636e\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251\uff1a\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Dyne \u9996\u5e2d\u533b\u7597\u5b98 Doug Kerr \u533b\u5b66\u535a\u58eb\u8868\u793a\uff1a\u201cDYNE-251 \u83b7\u5f97\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\uff0c\u8bc1\u660e\u4e86\u5176\u4f5c\u4e3a\u4e0b\u4e00\u4ee3\u7597\u6cd5\u7684\u6f5c\u529b\uff0c\u65e8\u5728\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u5e26\u6765\u6709\u610f\u4e49\u7684\u529f\u80fd\u6539\u5584\u3002\u5bf9\u4e8e\u8fd9\u4e9b\u60a3\u8005\uff0c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u53ef\u5bfc\u81f4\u4ea7\u751f\u8fd1\u4e4e\u5168\u957f\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\u3002\u6b63\u5982\u6211\u4eec\u4e4b\u524d\u62ab\u9732\u7684\uff0cDYNE-251 \u5df2\u8bc1\u660e\u5728 18 \u4e2a\u6708\u5185\u6301\u7eed\u4fdd\u6301\u529f\u80fd\u6539\u5584\uff0c\u8fd9\u901a\u8fc7\u8d77\u8eab\u65f6\u95f4\u548c\u6b65\u5e45\u901f\u5ea6\u7b2c 95 \u767e\u5206\u4f4d\u6570\u7b49\u5173\u952e\u6307\u6807\u6765\u8bc4\u4f30\u3002\u89c2\u5bdf\u5230\u7684\u8fd1\u4e4e\u5168\u957f\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\u8868\u8fbe\u6c34\u5e73\u6807\u5fd7\u7740\u6211\u4eec\u5411\u524d\u8fc8\u51fa\u4e86\u91cd\u8981\u4e00\u6b65\uff0c\u6709\u671b\u4e3a\u60a3\u8005\u5e26\u6765\u6709\u610f\u4e49\u7684\u76ca\u5904\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/exon-51-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5373\u5c06\u63a8\u51fa\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-receives-european-medicines-agency-ema-orphan-drug-designation-for-dyne-251-in-duchenne-muscular-dystrophy\/\">Dyne Therapeutics \u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 DYNE-251<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. The designation is based on data from the ongoing DELIVER clinical trial. DYNE-251: Breakthrough Therapy Designation \u201cThis Breakthrough Therapy Designation for DYNE-251 [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4275,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[208,145,122,193,194,88],"class_list":["post-4274","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dyne-therapeutics","tag-dyne-251","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-fda"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4274","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=4274"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4274\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4275"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4274"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4274"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4274"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}