{"id":3866,"date":"2025-05-19T13:43:11","date_gmt":"2025-05-19T10:43:11","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3866"},"modified":"2025-05-19T13:43:16","modified_gmt":"2025-05-19T10:43:16","slug":"genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/genethon-reports-results-of-gnt0004-gene-therapy-clinical-trial-permanent-decrease-in-ck-levels\/","title":{"rendered":"Genethon\u62a5\u544aGNT0004\u57fa\u56e0\u6cbb\u7597\u4e34\u5e8a\u8bd5\u9a8c\u7ed3\u679c\uff1aCK\u6c34\u5e73\u6c38\u4e45\u6027\u4e0b\u964d\u8d85\u8fc775%"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u5b89\u5168\u6027\u3001\u6709\u6548\u6027\u548c\u836f\u6548\u52a8\u529b\u5b66\u7ed3\u679c\u663e\u793a\uff0cGNT0004 \u4e0e\u77ed\u6682\u6027\u514d\u75ab\u9884\u9632\u6cbb\u7597\u76f8\u5173\u7684\u8010\u53d7\u6027\u826f\u597d\uff0c\u4e14\u5728\u5fae\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u3001CPK \u964d\u4f4e\u548c\u4e34\u5e8a\u6307\u6807\uff08NSAA\u3001\u5b9a\u65f6\u6d4b\u8bd5\uff09\u65b9\u9762\u5747\u8868\u73b0\u51fa\u826f\u597d\u7684\u7597\u6548\u6570\u636e\u3002\u63a5\u53d7\u6709\u6548\u5242\u91cf\u6cbb\u7597\u7684\u60a3\u8005\u8fd0\u52a8\u529f\u80fd\u5f97\u5230\u957f\u671f\u6539\u5584\u6216\u7a33\u5b9a\uff0c\u808c\u9178\u6fc0\u9176 (CPK) \u6c34\u5e73\u663e\u8457\u6301\u7eed\u964d\u4f4e\uff0c\u800c\u808c\u9178\u6fc0\u9176 (CPK) \u6c34\u5e73\u662f\u808c\u8089\u635f\u4f24\u7684\u5173\u952e\u6307\u6807\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">GNT0004\u4e34\u5e8a\u8bd5\u9a8c\u7684\u4e3b\u8981\u7ed3\u679c<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>GNT0004 \u4ea7\u54c1\u8bc1\u5b9e\u4e86\u5176\u4e34\u5e8a\u7597\u6548\uff0c\u5728\u6709\u6548\u5242\u91cf\u6cbb\u7597\u4e0b\u60a3\u8005\u7684\u8fd0\u52a8\u529f\u80fd\u53ef\u6301\u7eed\u7a33\u5b9a\u957f\u8fbe 2 \u5e74\u3002<\/li>\n\n\n\n<li>\u808c\u9178\u78f7\u9178\u6fc0\u9176\uff08CPK\uff09\u6c34\u5e73\u6301\u7eed\u663e\u8457\u964d\u4f4e\uff0c\u808c\u8089\u635f\u4f24\u7684\u751f\u7269\u6807\u5fd7\u7269\uff0c\u5728\u63a5\u53d7\u6709\u6548\u5242\u91cf\u6cbb\u7597\u7684 3 \u540d\u60a3\u8005\u4e2d\uff0c18 \u4e2a\u6708\u65f6\u5e73\u5747\u964d\u4f4e\u8d85\u8fc7 75%\u3002<\/li>\n\n\n\n<li>\u6b27\u6d32\u548c\u7f8e\u56fd\u8ba1\u5212\u4e8e 2025 \u5e74\u4e2d\u671f\u8fdb\u5165\u5173\u952e\u9636\u6bb5\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u6cbb\u7597\u4e00\u5e74\u540e\uff0c\u5bf9\u63a5\u53d7\u6709\u6548\u5242\u91cf\u6cbb\u7597\u7684\u4e09\u540d\u60a3\u8005\u4e0e\u5728\u540c\u4e00\u4e2d\u5fc3\u7531\u540c\u4e00\u533b\u751f\u968f\u8bbf\u7684\u5e74\u9f84\u5339\u914d\u7684\u4e00\u7ec4 34 \u540d\u672a\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\u8fdb\u884c\u6bd4\u8f83\uff0c\u7ed3\u679c\u663e\u793a\uff0c\u4f7f\u7528\u56fd\u9645\u516c\u8ba4\u7684\u4e34\u5e8a\u8bc4\u4f30\u91cf\u8868 NSAA \u83b7\u5f97\u7684\u8bc4\u5206\u5728\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\u548c\u672a\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\u4e4b\u95f4\u76f8\u5dee +4.7 \u5206\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u6cbb\u7597\u540e 24 \u4e2a\u6708\uff0c\u4e3b\u8981\u89c2\u5bdf\u7ed3\u679c\u5305\u62ec\uff1a<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5728\u63a5\u53d7\u6709\u6548\u5242\u91cf\u6cbb\u7597\u76843\u540d\u60a3\u8005\u4e2d\uff0c\u67092\u540d\u5728\u6cbb\u7597\u540e\u4e24\u5e74\u5185\u8fbe\u5230\u7597\u6548\uff0c\u8bd5\u9a8c\u663e\u793a\uff0c\u901a\u8fc7NSAA\u91cf\u8868\u6d4b\u91cf\uff0c\u8fd0\u52a8\u529f\u80fd\u5df2\u8d8b\u4e8e\u7a33\u5b9a\uff1b\u800c\u540c\u671f\u81ea\u7136\u53f2\u7814\u7a76\u4e2d\u672a\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\uff0cNSAA\u5e73\u5747\u6c34\u5e73\u6301\u7eed\u663e\u8457\u4e0b\u964d\u3002\u5176\u4e2d\u4e00\u540d\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\uff0c\u89c2\u5bdf\u5230\u7684\u6539\u5584\u4f7f\u5176\u572812\u4e2a\u6708\u65f6\u8fbe\u5230\u6700\u9ad8\u8bc4\u520634\u5206\uff0c\u5e76\u5728\u6cbb\u7597\u540e24\u4e2a\u6708\u5f97\u5230\u8bc1\u5b9e\u3002<\/li>\n\n\n\n<li>CPK \u964d\u4f4e\u5e73\u5747\u7a33\u5b9a\u5728 50% \u548c 87% \u4e4b\u95f4\uff1a\u6cbb\u7597\u540e 18 \u4e2a\u6708\u65f6 &gt;75%\uff08\u6765\u81ea\u4ee5\u6709\u6548\u5242\u91cf\u6cbb\u7597\u7684 3 \u540d\u60a3\u8005\u7684\u6570\u636e\uff09\uff0c\u5e76\u4e14\u6301\u7eed\u5b58\u5728\uff08\u5bf9\u4ee5\u6b64\u5242\u91cf\u6cbb\u7597\u7684\u524d\u4e24\u540d\u60a3\u8005\u8fdb\u884c\u957f\u8fbe 24 \u4e2a\u6708\u7684\u968f\u8bbf\uff09\u3002<\/li>\n\n\n\n<li>\u8be5\u57fa\u56e0\u6cbb\u7597\u836f\u7269\u7684\u5b89\u5168\u6027\u4ee4\u4eba\u653e\u5fc3\uff0c\u6ce8\u5c04\u4e24\u5e74\u540e\uff0c\u5728\u6240\u9009\u5242\u91cf\u4e0b\u6ca1\u6709\u53d1\u751f\u4e25\u91cd\u7684\u4e0d\u826f\u53cd\u5e94\uff0c\u660e\u663e\u4f4e\u4e8e\u6b63\u5728\u5f00\u53d1\u7684\u5176\u4ed6\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u4ea7\u54c1\u7684\u5242\u91cf\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a\uff1a<\/strong> <a href=\"https:\/\/dmdwarrior.com\/zh\/genethon-gnt0004-gene-therapy-duchenne-muscular-dystrophy\/\">\u5173\u4e8eGenethon\u57fa\u56e0\u7597\u6cd5GNT0004\u4f60\u5e94\u8be5\u77e5\u9053\u7684\u4e00\u5207<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of microdystrophin expression, CPK reduction, and clinical criteria (NSAA, timed tests). Patients treated at the effective dose show prolonged improvement or stabilization of motor functions and significant persistent reduction in creatine kinase [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3868,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[117,66,342,61,105,221,220],"class_list":["post-3866","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-dmd","tag-duchenne-muscular-dystrophy","tag-frederic-revah","tag-gene-therapy","tag-genethon","tag-gnt-0004-2","tag-gnt0004"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3866","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=3866"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3866\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3868"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3866"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3866"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3866"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}