{"id":5183,"date":"2025-11-19T18:44:39","date_gmt":"2025-11-19T15:44:39","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=5183"},"modified":"2025-11-19T19:06:37","modified_gmt":"2025-11-19T16:06:37","slug":"avidity-biosciences-announces-u-s-managed-access-program-map-for-investigational-therapy-del-zota-in-dmd44","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/avidity-biosciences-announces-u-s-managed-access-program-map-for-investigational-therapy-del-zota-in-dmd44\/","title":{"rendered":"Avidity Biosciences \u5ba3\u5e03\u5176\u5728\u7814\u7597\u6cd5 del-zota\uff08\u5916\u663e\u5b50 44 \u8df3\u8dc3\uff09\u5728\u7f8e\u56fd\u542f\u52a8\u7ba1\u7406\u51c6\u5165\u8ba1\u5212 (MAP)\u3002"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Avidity Biosciences \u5ba3\u5e03\u4e86\u5176\u5728\u7f8e\u56fd\u9488\u5bf9\u7b26\u5408\u6761\u4ef6\u7684\u3001\u60a3\u6709\u9002\u5408\u5916\u663e\u5b50 44 \u8df3\u8dc3 (DMD44) \u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7a81\u53d8\u60a3\u8005\u7684\u8bd5\u9a8c\u6027\u7597\u6cd5 delpacibart zotadirsen (del-zota) \u7684\u7ba1\u7406\u51c6\u5165\u8ba1\u5212 (MAP)\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u201c\u6211\u4eec\u6df1\u77e5DMD44\u60a3\u8005\u7fa4\u4f53\u9762\u4e34\u7740\u5de8\u5927\u7684\u9700\u6c42\uff0c\u56e0\u4e3a\u76ee\u524d\u5c1a\u65e0\u83b7\u6279\u7684\u9488\u5bf9\u8be5\u75be\u75c5\u7684\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\uff0c\u800cdel-zota\u57fa\u4e8e\u6211\u4eec\u4e34\u5e8a\u7814\u7a76\u4e2d\u524d\u6240\u672a\u6709\u7684\u6570\u636e\uff0c\u5c55\u73b0\u51fa\u5de8\u5927\u7684\u6f5c\u529b\uff0c\u201dAvidity\u603b\u88c1\u517c\u9996\u5e2d\u6267\u884c\u5b98Sarah Boyce\u8868\u793a\u3002\u201c\u6211\u4eec\u5f88\u9ad8\u5174\u542f\u52a8\u8fd9\u9879\u5feb\u901f\u5ba1\u6279\u7a0b\u5e8f\uff0c\u4ee5\u4fbf\u80fd\u591f\u4ee5\u5408\u89c4\u7684\u65b9\u5f0f\u5c3d\u5feb\u4e3a\u7b26\u5408\u6761\u4ef6\u7684\u60a3\u8005\u63d0\u4f9bdel-zota\u3002\u201d<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6839\u636e FDA \u6388\u6743\u7684\u6cbb\u7597\u65b9\u6848\uff0cAvidity \u5c06\u901a\u8fc7\u53c2\u4e0e\u7684\u533b\u7597\u673a\u6784\u4e3a\u7b26\u5408\u6761\u4ef6\u7684 DMD44 \u60a3\u8005\uff08\u7537\u5b69\u548c\u7537\u6027\uff09\u63d0\u4f9b del-zota \u6cbb\u7597\u3002\u9884\u8ba1\u5c06\u4e8e\u5e74\u5e95\u5f00\u59cb\u62db\u52df\uff0c\u53c2\u4e0e EXPLORE44-OLE \u7814\u7a76\u7684\u60a3\u8005\u5728\u5b8c\u6210\u4e24\u5e74\u6cbb\u7597\u540e\u53ef\u4ee5\u9009\u62e9\u8f6c\u5165 MAP \u7814\u7a76\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7acb\u5373\u67e5\u770b<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6211\u7684\u513f\u5b50\u9002\u5408\u63a5\u53d7\u5916\u663e\u5b5044\u8df3\u8dc3\u7597\u6cd5\u5417\uff1f<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u57282025\u5e7410\u6708\u4e3e\u884c\u7684BLA\u9884\u5ba1\u4f1a\u8bae\u4e0a\uff0cAvidity\u4e0eFDA\u5c31del-zota\u7684BLA\u7533\u8bf7\u8fbe\u6210\u4e00\u81f4\uff0c\u8ba1\u5212\u4e8e2026\u5e74\u63d0\u4ea4\u7533\u8bf7\u4ee5\u5bfb\u6c42\u52a0\u901f\u5ba1\u6279\u3002\u4e00\u65e6FDA\u83b7\u5f97\u6279\u51c6\u5e76\u4e0a\u5e02\uff0c\u5404\u65b9\u5c06\u8fc7\u6e21\u5230\u5546\u4e1a\u5316\u836f\u7269\u4f9b\u5e94\u3002.<a href=\"https:\/\/investors.aviditybiosciences.com\/2025-11-19-Avidity-Biosciences-Announces-U-S-Managed-Access-Program-MAP-for-Investigational-Therapy-del-zota-in-DMD44#print\" target=\"_blank\" rel=\"noopener\"><\/a><\/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>Avidity Biosciences announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States. &#8220;We recognize the considerable needs facing the DMD44 community given there are no approved exon skipping therapies for this disease as well as [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5185,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,397,129,141,127,246],"class_list":["post-5183","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-dmd44","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies"],"subtitle":"Avidity Biosciences \u5df2\u5728\u7f8e\u56fd\u542f\u52a8\u4e86\u4e00\u9879\u7ba1\u7406\u51c6\u5165\u8ba1\u5212 (MAP)\uff0c\u65e8\u5728\u4e3a\u60a3\u6709\u9002\u5408\u5916\u663e\u5b50 44 \u8df3\u8dc3 (DMD44) \u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7a81\u53d8\u7684\u60a3\u8005\u63d0\u4f9b\u7814\u7a76\u6027 delpacibart zotadirsen (del-zota)\u3002.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5183","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=5183"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/5183\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5185"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=5183"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=5183"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=5183"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}