{"id":6747,"date":"2026-03-12T11:43:48","date_gmt":"2026-03-12T08:43:48","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6747"},"modified":"2026-06-24T22:01:09","modified_gmt":"2026-06-24T19:01:09","slug":"del-zota-phase-3-safari44-clinical-trial-duchenne-muscular-dystrophy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/del-zota-phase-3-safari44-clinical-trial-duchenne-muscular-dystrophy\/","title":{"rendered":"Del-Zota \u548c SAFARI44 \u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 III \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff1a\u7814\u7a76\u8bbe\u8ba1\u3001\u6570\u636e\u548c\u4e34\u5e8a\u610f\u4e49"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 del-zota 3 \u671f SAFARI44 \u4e34\u5e8a\u8bd5\u9a8c\u4ee3\u8868\u7740\u5728\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u5f00\u53d1\u9488\u5bf9\u5916\u663e\u5b50 44 \u53ef\u6cbb\u7597\u57fa\u56e0\u7a81\u53d8\u7684\u9776\u5411\u6cbb\u7597\u65b9\u9762\u8fc8\u51fa\u4e86\u91cd\u8981\u4e00\u6b65\u3002. <\/strong>\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/mutations-and-deletions-amenable-to-exon-44-skipping-therapies-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u9002\u5408\u5916\u663e\u5b5044\u8df3\u8dc3\u7597\u6cd5\u7684\u7a81\u53d8\u548c\u7f3a\u5931<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a\u4e34\u5e8a\u4e0e\u79d1\u5b66\u4f1a\u8bae\u4e0a\u53d1\u8868\u7684 SAFARI44 \u7814\u7a76\u8bc4\u4f30\u4e86 del-zota \u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\uff0cdel-zota \u662f\u7531 Avidity Biosciences \u5f00\u53d1\u7684\u4e00\u79cd\u7814\u7a76\u6027\u6297\u4f53\u5be1\u6838\u82f7\u9178\u5076\u8054\u7269 (AOC) \u7597\u6cd5\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>3 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u5efa\u7acb\u5728\u4ee4\u4eba\u9f13\u821e\u7684\u65e9\u671f\u6570\u636e\u4e4b\u4e0a\uff0c\u8fd9\u4e9b\u6570\u636e\u8868\u660e del-zota \u53ef\u4ee5\u663e\u8457\u6539\u5584\u7531 44 \u53f7\u5916\u663e\u5b50\u7a81\u53d8\u5f15\u8d77\u7684 DMD \u60a3\u8005\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u751f\u6210\u548c\u808c\u8089\u529f\u80fd\u3002.<\/strong><\/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=\"#key-highlights-of-the-safari-44-phase-3-trial\">SAFARI44 \u4e09\u671f\u4e34\u5e8a\u8bd5\u9a8c\u662f\u4ec0\u4e48\uff1f<\/a><\/li><li><a href=\"#primary-and-secondary-endpoints\">\u4e3b\u8981\u548c\u6b21\u8981\u7ec8\u70b9<\/a><\/li><li><a href=\"#mechanism-of-del-zota-therapy\">Del-Zota\u7597\u6cd5\u7684\u4f5c\u7528\u673a\u5236<\/a><\/li><li><a href=\"#earlier-clinical-data-explore-44-trial\">\u65e9\u671f\u4e34\u5e8a\u6570\u636e\uff1aEXPLORE44 \u4e34\u5e8a\u8bd5\u9a8c<\/a><ul><li><a href=\"#key-findings-from-explore-44\">EXPLORE44\u7684\u4e3b\u8981\u53d1\u73b0<\/a><\/li><\/ul><\/li><li><a href=\"#open-label-extension-ole-results\">\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u7814\u7a76\uff08OLE\uff09\u7ed3\u679c<\/a><\/li><li><a href=\"#safety-and-tolerability\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027<\/a><\/li><li><a href=\"#why-the-safari-44-trial-matters\">SAFARI44\u4e34\u5e8a\u8bd5\u9a8c\u4e3a\u4f55\u5982\u6b64\u91cd\u8981<\/a><\/li><li><a href=\"#in-which-countries-will-the-del-zota-phase-3-safari-44-clinical-trials-begin\">Del-Zota 3\u671f\u548cSAFARI44\u4e34\u5e8a\u8bd5\u9a8c\u5c06\u5728\u54ea\u4e9b\u56fd\u5bb6\u5f00\u59cb\uff1f<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"key-highlights-of-the-safari-44-phase-3-trial\" class=\"wp-block-heading\">SAFARI44 \u4e09\u671f\u4e34\u5e8a\u8bd5\u9a8c\u662f\u4ec0\u4e48\uff1f<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u5927\u7ea6\u6709 70 \u540d 2 \u5c81\u53ca\u4ee5\u4e0a\u7684\u53ef\u81ea\u4e3b\u884c\u8d70\u7684\u60a3\u8005\u5c06\u53c2\u4e0e\u3002.<\/strong><\/li>\n\n\n\n<li>\u53c2\u4e0e\u8005\u5c06\u6bcf\u516d\u5468\u63a5\u53d7\u4e00\u6b21\u9759\u8109\u6ce8\u5c04\uff0c\u6301\u7eed 54 \u5468\u3002.<\/li>\n\n\n\n<li><strong>\u8be5\u4e34\u5e8a\u8bd5\u9a8c\u5305\u62ec\uff1a<\/strong>\n<ul class=\"wp-block-list\">\n<li>\u968f\u673a\u53cc\u76f2\u6cbb\u7597\u9636\u6bb5\uff0854\u5468\uff09<\/li>\n\n\n\n<li><strong>\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u7814\u7a76\uff0854\u5468\uff09<\/strong><\/li>\n\n\n\n<li>\u5b89\u5168\u6027\u968f\u8bbf\uff086\u5468\uff09<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li><strong>\u53c2\u4e0e\u8005\u5c06\u6309 1:1 \u968f\u673a\u5206\u7ec4\u63a5\u53d7 del-zota 5 mg\/kg PMO\uff0828 mg\/kg \u603b AOC \u4f53\u91cd\uff09\u6216\u5b89\u6170\u5242\u3002.<\/strong><\/li>\n\n\n\n<li>\u5728\u53cc\u76f2\u6cbb\u7597\u671f\u95f4\uff0c\u603b\u5171\u5c06\u8fdb\u884c\u4e5d\u6b21\u8f93\u6db2\u3002.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8be5\u8bbe\u8ba1\u65e8\u5728\u786e\u5b9a del-zota \u662f\u5426\u80fd\u663e\u8457\u6539\u5584\u808c\u8089\u529f\u80fd\u548c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\uff0c\u8fd9\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5173\u952e\u6cbb\u7597\u9776\u70b9\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"primary-and-secondary-endpoints\" class=\"wp-block-heading\">\u4e3b\u8981\u548c\u6b21\u8981\u7ec8\u70b9<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>del-zota 3\u671f\u4e34\u5e8a\u8bd5\u9a8c\u548c SAFARI44 \u4e34\u5e8a\u8bd5\u9a8c\u7684\u4e3b\u8981\u7ec8\u70b9\u662f\uff1a<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e0a\u5347\u901f\u5ea6\u6240\u9700\u65f6\u95f4\u7684\u53d8\u5316<\/strong>\uff1a\u4e00\u79cd\u5e7f\u6cdb\u4f7f\u7528\u7684\u529f\u80fd\u6027\u6d4b\u91cf\u65b9\u6cd5\uff0c\u53cd\u6620\u4e0b\u80a2\u808c\u8089\u529b\u91cf\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6b21\u8981\u7ed3\u679c\u6307\u6807\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73<\/strong><\/li>\n\n\n\n<li><strong>\u56db\u7ea7\u53f0\u9636\u6500\u722c\u901f\u5ea6<\/strong><\/li>\n\n\n\n<li><strong>10\u7c73\u6b65\u884c\/\u8dd1\u6b65\u6d4b\u8bd5<\/strong><\/li>\n\n\n\n<li><strong>SV95C \u6b65\u5e45\u901f\u5ea6<\/strong><\/li>\n\n\n\n<li><strong>\u5317\u6781\u661f\u52a8\u6001\u8bc4\u4f30 (NSAA)<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u6307\u6807\u5e38\u7528\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\uff0c\u4ee5\u8bc4\u4f30\u75be\u75c5\u8fdb\u5c55\u548c\u529f\u80fd\u6539\u5584\u60c5\u51b5\u3002\u4e86\u89e3\u66f4\u591a\uff1a&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD\u4e2d\u7684\u751f\u7269\u6807\u5fd7\u7269<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"mechanism-of-del-zota-therapy\" class=\"wp-block-heading\">Del-Zota\u7597\u6cd5\u7684\u4f5c\u7528\u673a\u5236<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Del-zota \u662f\u4e00\u79cd\u6297\u4f53-\u5be1\u6838\u82f7\u9178\u5076\u8054\u7269 (AOC)\uff0c\u65e8\u5728\u5c06\u78f7\u9170\u4e8c\u80fa\u5417\u5549\u4ee3\u5be1\u805a\u4f53 (PMO) \u76f4\u63a5\u9012\u9001\u81f3\u808c\u8089\u7ec4\u7ec7\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7597\u6cd5\u7684\u76ee\u7684\u662f\uff1a<\/p>\n\n\n\n<ol class=\"wp-block-list\">\n<li><strong>\u8df3\u8fc7\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u4e2d\u7684\u7b2c44\u53f7\u5916\u663e\u5b50<\/strong><\/li>\n\n\n\n<li><strong>\u6062\u590d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767dmRNA\u7684\u9605\u8bfb\u6846<\/strong><\/li>\n\n\n\n<li><strong>\u4fc3\u8fdb\u4ea7\u751f\u4e00\u79cd\u66f4\u77ed\u4f46\u529f\u80fd\u6b63\u5e38\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002<\/strong><\/li>\n<\/ol>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bf9\u4e8e\u57fa\u56e0\u7a81\u53d8\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u7684\u60a3\u8005\uff0c\u8fd9\u79cd\u7b56\u7565\u53ef\u80fd\u6709\u52a9\u4e8e\u7a33\u5b9a\u808c\u8089\u7ea4\u7ef4\u5e76\u51cf\u7f13\u75be\u75c5\u8fdb\u5c55\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"earlier-clinical-data-explore-44-trial\" class=\"wp-block-heading\">\u65e9\u671f\u4e34\u5e8a\u6570\u636e\uff1aEXPLORE44 \u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>EXPLORE44 \u7814\u7a76\u5efa\u7acb\u5728 EXPLORE44 1\/2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7ed3\u679c\u4e4b\u4e0a\uff0c\u8be5\u8bd5\u9a8c\u7eb3\u5165\u4e86 26 \u540d\u60a3\u6709\u5916\u663e\u5b50 44 \u53ef\u6cbb\u7597\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u53c2\u4e0e\u8005\u3002.<\/strong><\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"968\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-968x1024.jpg\" alt=\"\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u53ef\u901a\u8fc7\u5916\u663e\u5b50 44 \u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u7a81\u53d8\u548c\u7f3a\u5931\" class=\"wp-image-5275\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-968x1024.jpg 968w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-284x300.jpg 284w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-768x813.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-1452x1536.jpg 1452w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-11x12.jpg 11w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-397x420.jpg 397w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-150x159.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-300x317.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-696x737.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping-1068x1130.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/11\/Deletions-Amenable-to-Exon-44-Skipping.jpg 1890w\" sizes=\"auto, (max-width: 968px) 100vw, 968px\" \/><\/figure>\n\n\n\n<h3 id=\"key-findings-from-explore-44\" class=\"wp-block-heading\">EXPLORE44\u7684\u4e3b\u8981\u53d1\u73b0<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e34\u5e8a\u6570\u636e\u8868\u660e\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u9aa8\u9abc\u808c\u4e2dPMO\u6d53\u5ea6\u7ea6\u4e3a200 nM<\/strong><\/li>\n\n\n\n<li><strong>~25% \u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73<\/strong><\/li>\n\n\n\n<li><strong>37% \u5916\u663e\u5b50 44 \u8df3\u8dc3\u589e\u52a0<\/strong><\/li>\n\n\n\n<li><strong>&gt;80% \u808c\u9178\u6fc0\u9176\u51cf\u5c11<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0e\u81ea\u7136\u53f2\u961f\u5217\u76f8\u6bd4\uff0c\u529f\u80fd\u6027\u7ed3\u679c\u4e5f\u663e\u793a\u51fa\u4ee4\u4eba\u9f13\u821e\u7684\u7ed3\u679c\u3002\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping\/\" target=\"_blank\" rel=\"noreferrer noopener\">Del-zota (EXPLORE44) \u53ef\u63d0\u4f9b\u63a5\u8fd1 30% \u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u91cf<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"open-label-extension-ole-results\" class=\"wp-block-heading\">\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u7814\u7a76\uff08OLE\uff09\u7ed3\u679c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5728\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u7814\u7a76\u4e2d\u7ee7\u7eed\u63a5\u53d7\u6cbb\u7597\u7684\u53c2\u4e0e\u8005\u5728 12-24 \u4e2a\u6708\u5185\u83b7\u5f97\u4e86\u53ef\u8861\u91cf\u7684\u529f\u80fd\u6027\u76ca\u5904\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u89c2\u5bdf\u5230\u7684\u7ed3\u679c\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u56db\u7ea7\u53f0\u9636\u6500\u722c\u6d4b\u8bd5<\/strong>\n<ul class=\"wp-block-list\">\n<li>\u63d0\u5347\u4e86 2.1 \u79d2<\/li>\n\n\n\n<li><strong>\u81ea\u7136\u53f2\u961f\u5217\u4e0b\u964d\u4e86 2.7 \u79d2<\/strong><\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>10\u7c73\u6b65\u884c\/\u8dd1\u6b65\n<ul class=\"wp-block-list\">\n<li><strong>\u6cbb\u7597\u7ec4\u6539\u55840.7\u79d2\uff0c\u800c\u672a\u6cbb\u7597\u7ec4\u4e0b\u964d1.5\u79d2<\/strong><\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>\u8be5\u4ece\u5730\u677f\u4e0a\u7ad9\u8d77\u6765\u4e86\n<ul class=\"wp-block-list\">\n<li><strong>\u63d0\u53473.2\u79d2\uff0c\u4e0b\u964d1.6\u79d2<\/strong><\/li>\n<\/ul>\n<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5176\u4ed6\u53d1\u73b0\u8fd8\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005NSAA\u8bc4\u5206\u7a33\u5b9a<\/strong><\/li>\n\n\n\n<li><strong>\u4e0a\u80a2\u529f\u80fd (PUL) \u8bc4\u5206\u63d0\u9ad8 1.5 \u5206<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u4e9b\u7ed3\u679c\u8868\u660e\uff0cdel-zota \u53ef\u80fd\u51cf\u7f13\u6216\u7a33\u5b9a\u529f\u80fd\u8870\u9000\uff0c\u8fd9\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6cbb\u7597\u7684\u4e00\u4e2a\u4e3b\u8981\u76ee\u6807\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"safety-and-tolerability\" class=\"wp-block-heading\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u65e9\u671f\u4e34\u5e8a\u7814\u7a76\u4e2d\uff0cdel-zota \u8868\u73b0\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u5927\u591a\u6570\u4e0d\u826f\u4e8b\u4ef6\u4e3a\u8f7b\u5ea6\u81f3\u4e2d\u5ea6\u3002<\/strong><\/li>\n\n\n\n<li>\u672a\u62a5\u544a\u4efb\u4f55\u610f\u5916\u5b89\u5168\u4fe1\u53f7\u3002<\/li>\n\n\n\n<li><strong>\u89c2\u5bdf\u5230\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u663e\u8457\u964d\u4f4e\u3002<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u4e9b\u53d1\u73b0\u652f\u6301\u7ee7\u7eed\u8fdb\u884c 3 \u671f SAFARI44 \u4e34\u5e8a\u8bd5\u9a8c\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 id=\"why-the-safari-44-trial-matters\" class=\"wp-block-heading\">SAFARI44\u4e34\u5e8a\u8bd5\u9a8c\u4e3a\u4f55\u5982\u6b64\u91cd\u8981<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9488\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684 del-zota 3 \u671f SAFARI44 \u4e34\u5e8a\u8bd5\u9a8c\u53ef\u80fd\u4e3a\u652f\u6301\u9488\u5bf9\u5916\u663e\u5b50 44 \u9002\u7528\u60a3\u8005\u7684\u65b0\u7cbe\u51c6\u7597\u6cd5\u63d0\u4f9b\u5173\u952e\u8bc1\u636e\u3002.<\/strong> \u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/upcoming-exon-44-skipping-therapies-for-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\u5b5044\u8df3\u8dc3\u7597\u6cd5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c\u6210\u529f\uff0cdel-zota \u53ef\u80fd\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u589e\u52a0\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f<\/li>\n\n\n\n<li>\u6539\u5584\u808c\u8089\u529f\u80fd<\/li>\n\n\n\n<li>\u5ef6\u7f13\u75be\u75c5\u8fdb\u5c55<\/li>\n\n\n\n<li>\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u7fa4\u4f53\u62d3\u5c55\u6cbb\u7597\u9009\u62e9<\/li>\n<\/ul>\n\n\n\n<h2 id=\"in-which-countries-will-the-del-zota-phase-3-safari-44-clinical-trials-begin\" class=\"wp-block-heading\">Del-Zota 3\u671f\u548cSAFARI44\u4e34\u5e8a\u8bd5\u9a8c\u5c06\u5728\u54ea\u4e9b\u56fd\u5bb6\u5f00\u59cb\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">del-zota \u7684 III \u671f\u4e34\u5e8a\u8bd5\u9a8c SAFARI44 \u662f\u4e00\u9879\u5168\u7403\u6027\u4e34\u5e8a\u7814\u7a76\uff0c\u8fd9\u610f\u5473\u7740\u53d7\u8bd5\u8005\u62db\u52df\u5c06\u5728\u591a\u4e2a\u5730\u533a\u8fdb\u884c\uff0c\u800c\u975e\u4ec5\u9650\u4e8e\u5355\u4e00\u56fd\u5bb6\u3002\u8be5\u7814\u7a76\u9884\u8ba1\u5c06\u5728\u6b27\u6d32\u3001\u4e9a\u6d32\u53ca\u5176\u4ed6\u5730\u533a\u7684\u591a\u4e2a\u56fd\u5bb6\u62db\u52df\u53d7\u8bd5\u8005\u3002Avidity Biosciences \u9884\u8ba1\u5c06\u516c\u5e03 SAFARI44 \u7684\u8bd5\u9a8c\u5f00\u5c55\u56fd\u5bb6\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>The del-zota phase 3 SAFARI44 clinical trial for Duchenne muscular dystrophy represents a major step toward developing targeted treatments for patients with exon-44\u2013amenable genetic mutations in Duchenne Muscular Dystrophy. Learn More: Mutations and Deletions Amenable to Exon 44 Skipping Therapies Presented at the Muscular Dystrophy Association Clinical &amp; Scientific Conference, the SAFARI44 study evaluates the [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6751,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[126,137,298,141,127,246,77,178],"class_list":["post-6747","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-avidity-biosciences","tag-clinical-trial","tag-del-zota","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-exon-skipping","tag-exon-skipping-therapies"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6747","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=6747"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6747\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/6751"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=6747"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=6747"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=6747"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}