{"id":3720,"date":"2025-04-18T14:28:26","date_gmt":"2025-04-18T11:28:26","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3720"},"modified":"2025-04-18T14:39:11","modified_gmt":"2025-04-18T11:39:11","slug":"del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/del-zota-explore44-provides-nearly-30-normal-dystrophin-production-in-patients-amenable-to-exon-44-skipping\/","title":{"rendered":"Del-zota\uff08EXPLORE44\uff09\u53ef\u4e3a\u63a5\u53d7\u5916\u663e\u5b5044\u8df3\u8dc3\u6cbb\u7597\u7684\u60a3\u8005\u63d0\u4f9b\u63a5\u8fd130%\u7684\u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u751f\u6210"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u76ee\u524d\u6b63\u5728\u8fdb\u884c\u4e00\u9879 1\/2 \u671f EXPLORE44 \u7814\u7a76 (NCT05670730)\uff0c\u4ee5\u8bc4\u4f30 Del-zota\uff0c\u8be5\u836f\u7269\u7684\u5f00\u53d1\u76ee\u7684\u662f\u6062\u590d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u9605\u8bfb\u6846\u67b6\u5e76\u4ea7\u751f\u5177\u6709\u529f\u80fd\u6027\u4e14\u5185\u90e8\u622a\u77ed\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">2025\u5e74\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a(MDA)\u4e34\u5e8a\u4e0e\u79d1\u5b66\u5927\u4f1a\u4e8e3\u670816\u65e5\u81f319\u65e5\u5728\u5fb7\u514b\u8428\u65af\u5dde\u8fbe\u62c9\u65af\u4e3e\u884c\uff0c\u5927\u4f1a\u4e0a\u516c\u5e03\u4e86\u8fd9\u9879\u968f\u673a\u3001\u53cc\u76f2\u3001\u5b89\u6170\u5242\u5bf9\u7167\u8bd5\u9a8c\u7684\u7ed3\u679c\u3002\u7814\u7a76\u7ed3\u679c\u8868\u660e\uff0cDel-zota\u80fd\u591f\u4fc3\u8fdb\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u751f\u6210\u548c\u5916\u663e\u5b50\u8df3\u8dc3\uff0c\u540c\u65f6\u964d\u4f4e\u808c\u9178\u6fc0\u9176(CK)\u7684\u8ba1\u6570\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Avidity \u9996\u5e2d\u79d1\u5b66\u5b98 Michael Flanagan \u535a\u58eb\u63a5\u53d7\u4e86 NeurologyLive \u7684\u91c7\u8bbf\uff0c\u8be6\u7ec6\u8ba8\u8bba\u4e86 Del-zota \u53ca\u5176 1\/2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u7ed3\u679c\u3002\u4ed6\u89e3\u91ca\u4e86\u9488\u5bf9\u5916\u663e\u5b50 44 \u7684\u72ec\u7279\u7ed9\u836f\u65b9\u5f0f\uff0c\u4ee5\u53ca\u5b83\u4e0e\u5df2\u83b7\u6279\u548c\u6b63\u5728\u7814\u7a76\u7684\u5176\u4ed6\u836f\u7269\u7684\u4e0d\u540c\u4e4b\u5904\u3002Flanagan \u8fd8\u9884\u544a\u4e86\u8be5\u836f\u7269\u7684\u672a\u6765\u8ba1\u5212\uff0c\u5305\u62ec\u4e00\u9879\u9a8c\u8bc1\u6027 3 \u671f\u4e34\u5e8a\u8bd5\u9a8c\u3001\u4e00\u9879\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u8bd5\u9a8c\u4ee5\u53ca\u4e00\u9879\u53ef\u80fd\u7684\u751f\u7269\u5236\u5242\u8bb8\u53ef\u8bc1\u7533\u8bf7\u3002<\/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=\"#phase-1-2-explore-44-trial-design\">\u7b2c 1\/2 \u9636\u6bb5\u63a2\u7d22 44 \u8bd5\u9a8c\u8bbe\u8ba1<\/a><\/li><li><a href=\"#safety-and-tolerability\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027<\/a><\/li><li><a href=\"#biomarker-outcomes\">\u751f\u7269\u6807\u5fd7\u7269\u7ed3\u679c<\/a><\/li><li><a href=\"#creatine-kinase-ck-levels\">\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"phase-1-2-explore-44-trial-design\">\u7b2c 1\/2 \u9636\u6bb5\u63a2\u7d22 44 \u8bd5\u9a8c\u8bbe\u8ba1<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u968f\u673a\u3001\u5b89\u6170\u5242\u5bf9\u7167\u3001\u53cc\u76f2\u8bd5\u9a8c<\/li>\n\n\n\n<li>24 \u540d\u60a3\u6709 DMD44 \u7684\u53c2\u4e0e\u8005\uff08\u5e74\u9f84 7-27 \u5c81\uff0c\u5305\u62ec\u53ef\u884c\u8d70\u548c\u4e0d\u53ef\u884c\u8d70\u8005\uff09<\/li>\n\n\n\n<li>\u6cbb\u7597\u7ec4\uff1a\u6bcf 6 \u5468 5 mg\/kg vs \u6bcf 8 \u5468 10 mg\/kg vs \u5b89\u6170\u5242\uff083:1 \u968f\u673a\u5316\uff09<\/li>\n\n\n\n<li>\u5728\u57fa\u7ebf\u548c\u7b2c\u4e09\u6b21\u7ed9\u836f\u540e\u4e00\u4e2a\u6708\u8fdb\u884c\u808c\u8089\u6d3b\u68c0<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"safety-and-tolerability\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5b89\u5168\u6027\u826f\u597d\uff0c\u5927\u591a\u6570\u4e0d\u826f\u4e8b\u4ef6\u4e3a\u8f7b\u5ea6\u81f3\u4e2d\u5ea6<\/li>\n\n\n\n<li>\u6700\u5e38\u89c1\u7684\u4e0d\u826f\u4e8b\u4ef6\uff1a\u624b\u672f\u75bc\u75db\u548c\u5934\u75db<\/li>\n\n\n\n<li>\u4e24\u540d\u53c2\u4e0e\u8005\u505c\u6b62\u6cbb\u7597\uff1a1 \u540d\u56e0\u8fc7\u654f\u53cd\u5e94\uff0c1 \u540d\u56e0\u4e2d\u5ea6\u8f93\u6db2\u76f8\u5173\u53cd\u5e94<\/li>\n\n\n\n<li>\u65e0\u75c7\u72b6\u6027\u8840\u7ea2\u86cb\u767d\u53d8\u5316\uff0c\u65e0\u4f4e\u9541\u8840\u75c7\uff0c\u65e0\u6b7b\u4ea1<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"biomarker-outcomes\">\u751f\u7269\u6807\u5fd7\u7269\u7ed3\u679c<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u4e24\u4e2a\u5242\u91cf\u7ec4\u7684 PMO \u808c\u8089\u6d53\u5ea6\u5747\u4fdd\u6301\u7a33\u5b9a\u4e14\u8f83\u9ad8<\/li>\n\n\n\n<li>\u5916\u663e\u5b5044\u53d1\u751f\u663e\u8457\u8df3\u8dc3\uff1a5 mg\/kg\u7ec4\u4e3a37%\uff0c10 mg\/kg\u7ec4\u4e3a43%<\/li>\n\n\n\n<li>\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u91cf\u5e73\u5747\u589e\u52a0\u5230\u6b63\u5e38\u6c34\u5e73\u7684\u7ea6 25%<\/li>\n\n\n\n<li>\u5728\u6240\u6709\u5916\u663e\u5b50 44 \u9002\u5e94\u57fa\u56e0\u578b\u4e2d\u89c2\u5bdf\u5230\u9ad8\u8fbe 58% \u7684\u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"creatine-kinase-ck-levels\">\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u63a5\u53d7\u6cbb\u7597\u7684\u53d7\u8bd5\u8005\u7684 CK \u6c34\u5e73\u6301\u7eed\u964d\u4f4e\u81f3\u63a5\u8fd1\u6b63\u5e38\u6c34\u5e73<\/li>\n\n\n\n<li>5 mg\/kg \u7ec4 70-140 \u5929\u548c 10 mg\/kg \u7ec4 84-140 \u5929\u7684 CK \u964d\u4f4e\u6301\u7eed\u63a5\u8fd1\u6b63\u5e38\u4e0a\u9650<\/li>\n\n\n\n<li>\u4e0e\u57fa\u7ebf\u76f8\u6bd4\uff0cCK \u6c34\u5e73\u964d\u4f4e\u8d85\u8fc7 80%<\/li>\n\n\n\n<li>\u5728\u5f00\u653e\u6807\u7b7e\u6269\u5c55\u4e2d\uff0c\u51cf\u91cf\u6301\u7eed\u957f\u8fbe 1 \u5e74<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/next-generation-exon-skipping-therapies-for-duchenne-muscular-dystrophy\/\">\u4e0b\u4e00\u4ee3\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>A phase 1\/2 EXPLORE44 study (NCT05670730) is now being conducted to evaluate Del-zota, which was developed with the intention of restoring the dystrophin reading frame and producing a dystrophin protein that is functional and internally truncated. At the 2025 Muscular Dystrophy Association (MDA) Clinical &amp; Scientific Conference, which took place in Dallas, Texas, from March [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3722,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[126,298,141,127,246,299],"class_list":["post-3720","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-avidity-biosciences","tag-del-zota","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-explore44"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3720","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=3720"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3720\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3722"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3720"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3720"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3720"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}