{"id":6739,"date":"2026-03-24T15:16:30","date_gmt":"2026-03-24T12:16:30","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=6739"},"modified":"2026-03-24T15:18:59","modified_gmt":"2026-03-24T12:18:59","slug":"inspire-duchenne-sgt-003-clinical-trial-update","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/inspire-duchenne-sgt-003-clinical-trial-update\/","title":{"rendered":"SGT-003\u57fa\u56e0\u7597\u6cd5\uff1aINSPIRE DUCHENNE III\u671f\u7814\u7a76\u542f\u52a8"},"content":{"rendered":"<p class=\"wp-block-paragraph\"><strong>Solid Biosciences \u516c\u53f8\u62a5\u544a\u4e86\u5176\u6b63\u5728\u8fdb\u884c\u7684 1\/2 \u671f INSPIRE DUCHENNE \u4e34\u5e8a\u8bd5\u9a8c\u7684\u4ee4\u4eba\u9f13\u821e\u7684\u4e2d\u671f\u7ed3\u679c\uff0c\u8be5\u8bd5\u9a8c\u65e8\u5728\u8bc4\u4f30\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u7814\u7a76\u6027\u57fa\u56e0\u7597\u6cd5 SGT-003\u3002.<\/strong> \u6570\u636e\u663e\u793a\uff0c\u8be5\u7597\u6cd5\u53ef\u80fd\u6709\u52a9\u4e8e\u6062\u590d\u5173\u952e\u808c\u8089\u86cb\u767d\uff0c\u6539\u5584\u808c\u8089\u5065\u5eb7\u751f\u7269\u6807\u5fd7\u7269\uff0c\u5e76\u7a33\u5b9a\u63a5\u53d7\u6cbb\u7597\u8005\u7684\u5fc3\u810f\u529f\u80fd\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u66f4\u65b0\u5728 2026 \u5e74\u808c\u8089\u840e\u7f29\u75c7\u534f\u4f1a (MDA) \u5e74\u4f1a\u4e0a\u53d1\u5e03\uff0c\u91cd\u70b9\u4ecb\u7ecd\u4e86\u808c\u8089\u6d3b\u68c0\u3001\u8840\u6db2\u751f\u7269\u6807\u5fd7\u7269\u3001\u5fc3\u810f\u8bc4\u4f30\u548c\u5b89\u5168\u76d1\u6d4b\u65b9\u9762\u7684\u65b0\u53d1\u73b0\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">INSPIRE DUCHENNE\u8bd5\u9a8c\u6982\u8ff0<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>INSPIRE DUCHENNE \u662f\u4e00\u9879\u9996\u6b21\u4eba\u4f53 1\/2 \u671f\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u65e8\u5728\u8bc4\u4f30 SGT-003 \u5728\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u513f\u7ae5\u4e2d\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u751f\u7269\u6d3b\u6027\u3002.<\/strong> \u7acb\u5373\u9605\u8bfb\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/faq-solid-biosciences-sgt-003-gene-therapy\/\" target=\"_blank\" data-type=\"link\" data-id=\"https:\/\/dmdwarrior.com\/faq-solid-biosciences-sgt-003-gene-therapy\/\" rel=\"noreferrer noopener\">\u5173\u4e8e Solid Biosciences SGT-003 \u57fa\u56e0\u6cbb\u7597\u7684\u5e38\u89c1\u95ee\u9898<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7814\u7a76\u7684\u4e3b\u8981\u7279\u70b9\u5305\u62ec\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u5355\u5242\u91cf\u57fa\u56e0\u6cbb\u7597\u8f93\u6ce8<\/strong><\/li>\n\n\n\n<li>\u5242\u91cf\u6c34\u5e73\u4e3a 1E14 vg\/kg<\/li>\n\n\n\n<li><strong>\u5f00\u653e\u6807\u7b7e\u3001\u591a\u4e2d\u5fc3\u8bbe\u8ba1<\/strong><\/li>\n\n\n\n<li>\u8be5\u7814\u7a76\u5728\u7f8e\u56fd\u3001\u52a0\u62ff\u5927\u3001\u610f\u5927\u5229\u548c\u82f1\u56fd\u768415\u4e2a\u4e34\u5e8a\u4e2d\u5fc3\u8fdb\u884c\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u622a\u81f3 2026 \u5e74 3 \u6708 11 \u65e5\uff0c\u5171\u6709 40 \u540d\u53c2\u4e0e\u8005\u6536\u5230\u4e86 SGT-003\u3002.<\/strong><\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u89c2\u5bdf\u5230\u5f3a\u70c8\u7684\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u808c\u8089\u6d3b\u68c0\u7ed3\u679c\u663e\u793a\uff0c\u6cbb\u7597\u540e\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u663e\u8457\u589e\u5f3a\u3002.<\/strong> \u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u662f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u7f29\u77ed\u7248\u672c\uff0c\u65e8\u5728\u5e2e\u52a9\u7a33\u5b9a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7684\u808c\u8089\u7ec6\u80de\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7814\u7a76\u4eba\u5458\u8fd8\u89c2\u5bdf\u5230\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u76f8\u5173\u86cb\u767d\u590d\u5408\u7269 (DAPC) \u7684\u5173\u952e\u6210\u5206\u5f97\u5230\u6062\u590d\uff0c\u5305\u62ec\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u03b2-\u808c\u805a\u7cd6<\/strong><\/li>\n\n\n\n<li><strong>\u795e\u7ecf\u5143\u578b\u4e00\u6c27\u5316\u6c2e\u5408\u9176\uff08nNOS\uff09<\/strong><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u86cb\u767d\u8d28\u5728\u7ef4\u6301\u808c\u8089\u5b8c\u6574\u6027\u65b9\u9762\u53d1\u6325\u7740\u81f3\u5173\u91cd\u8981\u7684\u4f5c\u7528\u3002\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u7f3a\u5931\u4f1a\u7834\u574f\u8fd9\u79cd\u590d\u5408\u7269\u7684\u7a33\u5b9a\u6027\uff0c\u4ece\u800c\u5bfc\u81f4\u808c\u8089\u635f\u4f24\u548c\u9000\u5316\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">SGT-003 \u7684\u663e\u8457\u4e4b\u5904\u5728\u4e8e\u5176\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6784\u5efa\u4f53\u542b\u6709 R16\/R17 \u7ed3\u5408\u57df\uff0c\u8fd9\u6709\u52a9\u4e8e\u5c06 nNOS \u6b63\u786e\u5b9a\u4f4d\u5728\u808c\u8089\u7ec4\u7ec7\u4e2d\u2014\u2014\u8fd9\u662f\u8bb8\u591a\u5176\u4ed6\u57fa\u56e0\u6cbb\u7597\u8bbe\u8ba1\u4e2d\u6240\u6ca1\u6709\u7684\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u751f\u7269\u6807\u5fd7\u7269\u6539\u5584\u8868\u660e\u808c\u8089\u7ea4\u7ef4\u66f4\u5065\u5eb7<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7814\u7a76\u8fd8\u6d4b\u91cf\u4e86\u591a\u79cd\u4e0e\u808c\u8089\u635f\u4f24\u548c\u4fee\u590d\u76f8\u5173\u7684\u751f\u7269\u6807\u5fd7\u7269\u3002\u7ed3\u679c\u8868\u660e\uff0cSGT-003\u6cbb\u7597\u53ef\u80fd\u6709\u52a9\u4e8e\u7a33\u5b9a\u808c\u8089\u7ea4\u7ef4\u5e76\u51cf\u5c11\u6301\u7eed\u7684\u9000\u5316\u3002\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" data-type=\"post\" data-id=\"6725\" rel=\"noreferrer noopener\">\u751f\u7269\u6807\u5fd7\u7269<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8bc4\u4f30\u7684\u4e00\u4e2a\u91cd\u8981\u6307\u6807\u662f\u80da\u80ce\u808c\u7403\u86cb\u767d\u91cd\u94fe\uff08eMHC\uff09\u3002.<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>eMHC\u901a\u5e38\u5728\u80ce\u513f\u53d1\u80b2\u8fc7\u7a0b\u4e2d\u8868\u8fbe\u3002.<\/li>\n\n\n\n<li>\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0c\u5f53\u808c\u8089\u8bd5\u56fe\u4fee\u590d\u6301\u7eed\u7684\u635f\u4f24\u65f6\uff0c\u8fd9\u79cd\u75c7\u72b6\u4f1a\u518d\u6b21\u51fa\u73b0\u3002.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7814\u7a76\u4eba\u5458\u89c2\u5bdf\u5230\u7b2c 90 \u5929 eMHC \u9633\u6027\u7ea4\u7ef4\u5e73\u5747\u51cf\u5c11\u4e86 44%\uff0c\u8fd9\u8868\u660e\u8be5\u7597\u6cd5\u53ef\u80fd\u6709\u52a9\u4e8e\u4e2d\u65ad\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u5e38\u89c1\u7684\u808c\u8089\u9000\u5316\u548c\u518d\u751f\u5faa\u73af\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e9b\u751f\u7269\u6807\u5fd7\u7269\u7684\u6539\u5584\u8868\u660e\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u51cf\u5c11\u808c\u8089\u635f\u4f24<\/li>\n\n\n\n<li>\u808c\u8089\u7ea4\u7ef4\u7a33\u5b9a\u6027\u63d0\u9ad8<\/li>\n\n\n\n<li>\u808c\u8089\u5e72\u7ec6\u80de\u7684\u4fdd\u5b58<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">\u5fc3\u810f\u529f\u80fd\u7a33\u5b9a\u7684\u8ff9\u8c61<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5c3d\u7ba1\u6700\u521d\u7eb3\u5165\u5fc3\u810f\u6d4b\u91cf\u53ea\u662f\u4e3a\u4e86\u5b89\u5168\u76d1\u6d4b\uff0c\u4f46\u7814\u7a76\u4eba\u5458\u89c2\u5bdf\u5230\u6cbb\u7597\u540e\u5fc3\u810f\u529f\u80fd\u8d8b\u4e8e\u7a33\u5b9a\u5e76\u6709\u6240\u6539\u5584\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u91c7\u7528\u5de6\u5fc3\u5ba4\u5c04\u8840\u5206\u6570\uff08LVEF\uff09\u8bc4\u4f30\u5fc3\u810f\u529f\u80fd\uff0c\u8be5\u6307\u6807\u8861\u91cf\u5fc3\u810f\u6cf5\u8840\u7684\u6548\u7387\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5bf9\u4e8e\u6cbb\u7597\u524d\u5de6\u5fc3\u5ba4\u5c04\u8840\u5206\u6570\u5904\u4e8e\u4f4e\u6b63\u5e38\u6c34\u5e73\u7684\u53c2\u4e0e\u8005\u6765\u8bf4\uff0c\u6539\u5584\u6548\u679c\u5c24\u4e3a\u660e\u663e\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u4e00\u53d1\u73b0\u5c24\u4e3a\u91cd\u8981\uff0c\u56e0\u4e3a\u5fc3\u808c\u75c5\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u7684\u4e3b\u8981\u6b7b\u56e0\u4e4b\u4e00\u3002\u7814\u7a76\u8868\u660e\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u7ea6\u6709251\u540dTP157T\u60a3\u8005\u57286\u5c81\u65f6\u51fa\u73b0\u5fc3\u808c\u75c5\u75c7\u72b6\u3002<\/strong><\/li>\n\n\n\n<li>\u5927\u7ea659%\u572810\u5c81\u65f6\u51fa\u73b0\u75c7\u72b6<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\">\u5b89\u5168\u6027\u548c\u8010\u53d7\u6027<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u76ee\u524d\u6765\u770b\uff0cSGT-003 \u5df2\u5c55\u73b0\u51fa\u826f\u597d\u7684\u5b89\u5168\u6027\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u63a5\u53d7\u6cbb\u7597\u768440\u540d\u53c2\u4e0e\u8005\u4e2d\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u8be5\u7597\u6cd5\u603b\u4f53\u8010\u53d7\u6027\u826f\u597d\u3002<\/strong><\/li>\n\n\n\n<li>\u4e2d\u671f\u5206\u6790\u4e2d\u672a\u62a5\u544a\u4efb\u4f55\u91cd\u5927\u5b89\u5168\u95ee\u9898\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7597\u6cd5\u7684\u53e6\u4e00\u4e2a\u663e\u8457\u7279\u70b9\u662f\u5176\u7b80\u5316\u7684\u514d\u75ab\u7ba1\u7406\u65b9\u6cd5\u3002\u8be5\u7597\u6cd5\u91c7\u7528\u4ec5\u4f7f\u7528\u7c7b\u56fa\u9187\u7684\u9884\u9632\u6027\u514d\u75ab\u8c03\u8282\u65b9\u6848\uff0c\u4e0e\u67d0\u4e9b\u5176\u4ed6\u57fa\u56e0\u7597\u6cd5\u4e2d\u4f7f\u7528\u7684\u66f4\u590d\u6742\u7684\u65b9\u6848\u76f8\u6bd4\uff0c\u8fd9\u53ef\u80fd\u51cf\u8f7b\u6cbb\u7597\u8d1f\u62c5\u3002.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">SGT-003 \u57fa\u56e0\u7597\u6cd5\uff1aINSPIRE DUCHENNE III \u671f\u4e34\u5e8a\u8bd5\u9a8c (NCT06138639)<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u9879\u7814\u7a76\u6b63\u5728\u8c03\u67e5\u4e00\u79cd\u540d\u4e3aSGT-003\u7684\u57fa\u56e0\u7597\u6cd5\uff0c\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u60a3\u8005\u3002\u60a3\u8005\u5fc5\u987b\u7b26\u5408\u7279\u5b9a\u6761\u4ef6\u624d\u80fd\u53c2\u4e0e\u8fd9\u9879\u7814\u7a76\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/how-to-participate-in-clinical-trials-for-duchenne\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u6211\u7684\u5b69\u5b50\u5982\u4f55\u53c2\u52a0\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e34\u5e8a\u8bd5\u9a8c\uff1f<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\">\u8c01\u53ef\u4ee5\u53c2\u52a0\uff1f\uff08\u53c2\u4e0e\u6807\u51c6\uff09<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5e74\u9f84\u7ec4\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7b2c\u4e00\u7ec4\uff1a4\u5c81\u81f37\u5c81\u4ee5\u4e0b<\/li>\n\n\n\n<li>\u7b2c\u4e8c\u7ec4\uff1a7\u5c81\u81f312\u5c81\u4ee5\u4e0b<\/li>\n\n\n\n<li>\u7b2c\u4e09\u7ec4\uff1a\u51fa\u751f\u81f34\u5c81\u4ee5\u4e0b<\/li>\n\n\n\n<li>\u7b2c\u56db\u7ec4\uff1a12\u5c81\u81f318\u5c81\u4ee5\u4e0b<\/li>\n\n\n\n<li>\u7b2c\u4e94\u7ec4\uff1a10\u5c81\u81f318\u5c81\u4ee5\u4e0b<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6b65\u884c\u80fd\u529b\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7b2c 1\u30012 \u548c 4 \u7ec4\uff1a\u5fc5\u987b\u80fd\u591f\u884c\u8d70<\/li>\n\n\n\n<li>\u7b2c\u4e09\u7ec4\uff1a\u53ef\u4ee5\u662f\u6b65\u884c\u8005\u6216\u975e\u6b65\u884c\u8005<\/li>\n\n\n\n<li>\u7b2c\u4e94\u7ec4\uff1a\u76ee\u524d\u5fc5\u987b\u65e0\u6cd5\u884c\u8d70\uff0c\u4f46\u8fc7\u53bb\u5fc5\u987b\u80fd\u591f\u884c\u8d70\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u6b64\u5916\uff0c\u53c2\u4e0e\u8005\uff08\u5982\u9002\u7528\uff09\u5fc5\u987b\u80fd\u591f\u5728 30 \u79d2\u5185\u5b8c\u6210 10 \u7c73\u6b65\u884c\/\u8dd1\u6b65\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8bca\u65ad\u548c\u9057\u4f20\u5b66\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5fc5\u987b\u786e\u8bca\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7<\/li>\n\n\n\n<li>\u5fc5\u987b\u5177\u6709\u4e0e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u76f8\u7b26\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u7a81\u53d8\u3002<\/li>\n\n\n\n<li>\u5728\u67d0\u4e9b\u60c5\u51b5\u4e0b\uff0c\u53ef\u80fd\u9700\u8981\u8fdb\u884c\u808c\u8089\u6d3b\u68c0\u6765\u6d4b\u91cf\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u514d\u75ab\u72b6\u6001\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5fc5\u987b\u8fdb\u884cAAV\uff08\u75c5\u6bd2\u8f7d\u4f53\uff09\u6297\u4f53\u68c0\u6d4b\uff0c\u4e14\u7ed3\u679c\u4e3a\u9634\u6027\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7c7b\u56fa\u9187\u4f7f\u7528\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7b2c 1\u30012\u30014 \u548c 5 \u7ec4\uff1a\n<ul class=\"wp-block-list\">\n<li>\u5fc5\u987b\u575a\u6301\u6bcf\u65e5\u7a33\u5b9a\u7684\u7c7b\u56fa\u9187\u6cbb\u7597\u65b9\u6848\u81f3\u5c11 12 \u5468\u3002<\/li>\n\n\n\n<li>\u8fd9\u5305\u62ec\u6cfc\u5c3c\u677e\uff08\u81f3\u5c11 0.5 mg\/kg\/\u5929\uff09\u6216\u5730\u592b\u53ef\u7279\uff08\u81f3\u5c11 0.75 mg\/kg\/\u5929\uff09\u3002<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>\u7b2c 3 \u7ec4\uff1a\u65e0\u9700\u4f7f\u7528\u7c7b\u56fa\u9187<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4f53\u683c\u68c0\u67e5\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5fc5\u987b\u7b26\u540810\u7c73\u6b65\u884c\/\u8dd1\u6b65\u6d4b\u8bd5\u6807\u51c6<\/li>\n\n\n\n<li>\u5fc5\u987b\u7b26\u5408\u4ece\u8eba\u5367\u5230\u7ad9\u7acb\u6240\u9700\u65f6\u95f4\u7684\u6807\u51c6<\/li>\n\n\n\n<li>\u7b2c 5 \u7ec4\u8fd8\u5fc5\u987b\u6ee1\u8db3\u4e0a\u80a2\u529f\u80fd\u8981\u6c42\uff08PUL 2.0 \u6d4b\u8bd5\uff09\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5176\u4ed6\u8981\u6c42\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u4f53\u91cd\u5fc5\u987b\u572890\u516c\u65a4\u6216\u4ee5\u4e0b\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06138639\" target=\"_blank\" rel=\"noreferrer noopener\">NCT06138639<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h3 class=\"wp-block-heading\">\u54ea\u4e9b\u4eba\u4e0d\u80fd\u53c2\u52a0\uff1f\uff08\u6392\u9664\u6807\u51c6\uff09<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u7b26\u5408\u4ee5\u4e0b\u6761\u4ef6\u7684\u53c2\u4e0e\u8005\u5c06\u65e0\u6cd5\u53c2\u52a0\u672c\u7814\u7a76\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u8fc7\u53bb\u4e09\u4e2a\u6708\u5185\u4f7f\u7528\u8fc7\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8c03\u8282\u836f\u7269\u3002<\/li>\n\n\n\n<li>\u4e4b\u524d\u662f\u5426\u63a5\u53d7\u8fc7\u4efb\u4f55\u57fa\u56e0\u6cbb\u7597\uff08\u5df2\u6279\u51c6\u6216\u5b9e\u9a8c\u6027\uff09<\/li>\n\n\n\n<li>\u8fc7\u53bb3\u4e2a\u6708\u5185\u670d\u7528\u8fc7\u67d0\u4e9b\u836f\u7269\uff0c\u6216\u8005\u836f\u7269\u5c1a\u672a\u5b8c\u5168\u4ece\u4f53\u5185\u6e05\u9664\u3002<\/li>\n\n\n\n<li>\u5df2\u786e\u8bca\u7684 DMD \u4e34\u5e8a\u8bca\u65ad\u4e0e DMD \u57fa\u56e0\u4e2d\u4efb\u4f55\u7f3a\u5931\u7a81\u53d8\u3001\u4e0d\u53d8\u7a81\u53d8\u6216\u9884\u6d4b\u4e0d\u8868\u8fbe\u5916\u663e\u5b50 1 \u81f3 11\u3001\u5916\u663e\u5b50 42 \u81f3 45 \u6216\u5916\u663e\u5b50 57 \u81f3 69\uff08\u542b\uff09\u7684\u53d8\u5f02\u76f8\u5173\uff0c\u5e76\u7531\u57fa\u56e0\u62a5\u544a\u8bb0\u5f55\uff0c\u4e14\u7531\u8d5e\u52a9\u5546\u57fa\u56e0\u68c0\u6d4b\u786e\u8ba4\u3002.<\/li>\n<\/ul>\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>Solid Biosciences has reported encouraging interim results from its ongoing Phase 1\/2 INSPIRE DUCHENNE clinical trial, evaluating the investigational gene therapy SGT-003 for Duchenne muscular dystrophy (DMD). The data suggest the therapy may help restore critical muscle proteins, improve biomarkers of muscle health, and stabilize heart function in treated participants. The update was presented at [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":6740,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[61,419,524,110,170,109],"class_list":["post-6739","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-gene-therapy","tag-impact-duchenne","tag-inspire-duchenne","tag-sgt-003","tag-sgt-003-gene-therapy","tag-solid-biosciences"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6739","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=6739"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/6739\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/6740"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=6739"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=6739"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=6739"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}