{"id":4672,"date":"2025-09-29T16:14:48","date_gmt":"2025-09-29T13:14:48","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4672"},"modified":"2025-09-29T16:16:28","modified_gmt":"2025-09-29T13:16:28","slug":"dyne-therapeutics-receives-orphan-drug-designation-in-japan-for-dyne-251-in-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-receives-orphan-drug-designation-in-japan-for-dyne-251-in-duchenne-muscular-dystrophy\/","title":{"rendered":"Dyne Therapeutics \u5728\u65e5\u672c\u83b7\u5f97\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 DYNE-251 \u7684\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Dyne Therapeutics \u5ba3\u5e03\uff0c\u65e5\u672c\u539a\u751f\u52b3\u52a8\u7701 (MHLW) \u5df2\u6388\u4e88 DYNE-251 \u5b64\u513f\u836f\u8d44\u683c\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\uff0c\u8fd9\u4e9b\u60a3\u8005\u7684 DMD \u57fa\u56e0\u7a81\u53d8\u53ef\u901a\u8fc7 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u6cbb\u7597\u3002DYNE-251 \u6b63\u5728\u63a5\u53d7\u6b63\u5728\u8fdb\u884c\u7684 1\/2 \u671f DELIVER \u5168\u7403\u4e34\u5e8a\u8bd5\u9a8c\u7684\u8bc4\u4f30\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u663e\u8457\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Dyne \u9996\u5e2d\u533b\u7597\u5b98 Doug Kerr \u533b\u5b66\u535a\u58eb\u8868\u793a\uff1a\u201c\u5728 DELIVER \u8bd5\u9a8c\u4e2d\uff0c\u6211\u4eec\u770b\u5230\u4e86\u524d\u6240\u672a\u6709\u7684\u6301\u7eed\u529f\u80fd\u6539\u5584\uff0c\u8fd9\u662f\u7531\u663e\u8457\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u63a8\u52a8\u7684\uff0c\u8fd9\u51f8\u663e\u4e86 DYNE-251 \u6539\u53d8\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u62a4\u7406\u7684\u6f5c\u529b\u3002\u201d <\/strong>DYNE-251 \u73b0\u5df2\u5728\u65e5\u672c\u83b7\u5f97\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a\uff0c\u8865\u5145\u4e86\u5176\u5728\u7f8e\u56fd\u548c\u6b27\u6d32\u7684\u73b0\u6709\u8d44\u683c\u8ba4\u5b9a\uff0c\u4f5c\u4e3a\u4e00\u9879\u524d\u666f\u5149\u660e\u7684\u4e0b\u4e00\u4ee3 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\uff0c\u5176\u53d1\u5c55\u524d\u666f\u5c06\u6301\u7eed\u770b\u597d\u3002\u643a\u5e26 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u7a81\u53d8\u7684\u4e2a\u4f53\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u60a3\u8005\u4e2d\u6700\u5e38\u89c1\u4e14\u529f\u80fd\u53d7\u5f71\u54cd\u6700\u4e25\u91cd\u7684\u7fa4\u4f53\u4e4b\u4e00\u3002\u89e3\u51b3\u8fd9\u4e00\u5c1a\u672a\u5f97\u5230\u6ee1\u8db3\u7684\u9700\u6c42\u6709\u671b\u8fdb\u4e00\u6b65\u63a8\u52a8\u6211\u4eec\u81f4\u529b\u4e8e\u5f00\u53d1\u4e00\u79cd\u80fd\u591f\u6539\u5584\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u529f\u80fd\u7684\u65b9\u6cd5\u3002\u6211\u4eec\u671f\u5f85\u4e0e\u65e5\u672c\u76d1\u7ba1\u673a\u6784\u5408\u4f5c\uff0c\u5171\u540c\u786e\u5b9a DYNE-251 \u5728\u65e5\u672c\u7684\u76d1\u7ba1\u9014\u5f84\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">DYNE-251\u5728\u65e5\u672c\u83b7\u5f97\u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5728\u65e5\u672c\uff0c\u5b64\u513f\u836f\u8d44\u683c\u6388\u4e88\u7528\u4e8e\u6cbb\u7597\u5f71\u54cd\u8be5\u56fd\u4e0d\u5230 50,000 \u540d\u60a3\u8005\u4e14\u533b\u7597\u9700\u6c42\u5f88\u9ad8\u7684\u7f55\u89c1\u75be\u75c5\u7684\u836f\u7269\u3002<\/strong> \u5176\u4f18\u52bf\u5305\u62ec\u5f00\u53d1\u6210\u672c\u8865\u8d34\u548c\u83b7\u6279\u540e\u957f\u8fbe 10 \u5e74\u7684\u6f5c\u5728\u5e02\u573a\u72ec\u5360\u6743\u3002DYNE-251 \u8fd8\u83b7\u5f97\u4e86\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u6388\u4e88\u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u3001\u5feb\u901f\u901a\u9053\u548c\u7f55\u89c1\u513f\u79d1\u75be\u75c5\u8ba4\u5b9a (FDA)\uff0c\u4ee5\u53ca FDA \u548c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u6388\u4e88\u7684\u5b64\u513f\u836f\u8ba4\u5b9a (EMA)\uff0c\u7528\u4e8e\u6cbb\u7597\u9002\u5408\u5916\u663e\u5b50 51 \u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u60a3\u8005\u3002 <strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-announces-fda-breakthrough-therapy-designation-for-dyne-251-in-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">DYNE-251 \u83b7\u5f97\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6211\u7684\u513f\u5b50\u9002\u5408\u63a5\u53d7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u5417\uff1f<\/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<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\">\u5173\u4e8eDYNE-251<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u662f\u4e00\u79cd\u5728\u7814\u6cbb\u7597\u836f\u7269\uff0c\u76ee\u524d\u6b63\u5728\u8fdb\u884c 1\/2 \u671f\u5168\u7403 DELIVER \u4e34\u5e8a\u8bd5\u9a8c\uff0c\u8be5\u8bd5\u9a8c\u9488\u5bf9\u643a\u5e26 DMD \u57fa\u56e0\u7a81\u53d8\u4e14\u53ef\u901a\u8fc7 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u60a3\u8005\u3002DYNE-251 \u7531\u78f7\u4e8c\u9170\u80fa\u5417\u5549\u5be1\u805a\u4f53 (PMO) \u4e0e\u6297\u539f\u7ed3\u5408\u7247\u6bb5 (Fab) \u5076\u8054\u800c\u6210\uff0c\u8be5\u7247\u6bb5\u53ef\u4e0e\u8f6c\u94c1\u86cb\u767d\u53d7\u4f53 1 (TfR1) \u7ed3\u5408\u3002\u5b83\u65e8\u5728\u4fc3\u8fdb\u808c\u8089\u548c\u4e2d\u67a2\u795e\u7ecf\u7cfb\u7edf (CNS) \u4ea7\u751f\u63a5\u8fd1\u5168\u957f\u7684\u6297\u808c\u840e\u7f29\u86cb\u767d\uff0c\u4ece\u800c\u6539\u5584\u529f\u80fd\u3002DYNE-251 \u5df2\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40 (FDA) \u7684\u7a81\u7834\u6027\u7597\u6cd5\u8ba4\u5b9a\u3001\u5feb\u901f\u901a\u9053\u548c\u513f\u79d1\u7f55\u89c1\u75be\u75c5\u8ba4\u5b9a (FDA)\uff0c\u4ee5\u53ca FDA \u548c\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40 (EMA) \u7684\u5b64\u513f\u836f\u8ba4\u5b9a\uff0c\u7528\u4e8e\u6cbb\u7597\u53ef\u901a\u8fc7 51 \u53f7\u5916\u663e\u5b50\u8df3\u8dc3\u6cbb\u7597\u7684 DMD \u60a3\u8005\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9664\u4e86 DYNE-251 \u4e4b\u5916\uff0cDyne \u8fd8\u5728\u5efa\u7acb DMD \u7279\u8bb8\u7ecf\u8425\u6743\uff0c\u5e76\u6709\u9488\u5bf9\u5176\u4ed6\u5916\u663e\u5b50\uff08\u5305\u62ec 53\u300145 \u548c 44\uff09\u7684\u4e34\u5e8a\u524d\u9879\u76ee\u3002<\/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\/\">\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>","protected":false},"excerpt":{"rendered":"<p>Dyne Therapeutics announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. DYNE-251 is being evaluated in the ongoing Phase 1\/2 DELIVER global clinical trial. [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4673,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[143,208,145,122,193,421,194,142],"class_list":["post-4672","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-japan"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4672","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=4672"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4672\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/4673"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4672"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4672"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4672"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}