{"id":3429,"date":"2025-03-31T12:19:41","date_gmt":"2025-03-31T09:19:41","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3429"},"modified":"2025-11-03T20:22:47","modified_gmt":"2025-11-03T17:22:47","slug":"exon-51-skipping-therapies-dmd","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/exon-51-skipping-therapies-dmd\/","title":{"rendered":"\u5373\u5c06\u63a8\u51fa\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u662f\u4e00\u79cd\u521b\u65b0\u7597\u6cd5\uff0c\u65e8\u5728\u89e3\u51b3\u5bfc\u81f4\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u57fa\u56e0\u7a81\u53d8\u3002\u901a\u8fc7\u8df3\u8dc3\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u4e2d\u7684\u5916\u663e\u5b50 51\uff0c\u8fd9\u4e9b\u7597\u6cd5\u6709\u52a9\u4e8e\u4ea7\u751f\u66f4\u77ed\u3001\u529f\u80fd\u6027\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u4ece\u800c\u6709\u53ef\u80fd\u51cf\u7f13\u75be\u75c5\u8fdb\u5c55\u5e76\u6539\u5584\u60a3\u8005\u7684\u751f\u6d3b\u8d28\u91cf\u3002\u8fd9\u79cd\u5c16\u7aef\u65b9\u6cd5\u4e3a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\u5e26\u6765\u4e86\u5e0c\u671b\uff0c\u91cd\u70b9\u662f\u589e\u5f3a\u808c\u8089\u529f\u80fd\u548c\u5ef6\u957f\u6d3b\u52a8\u80fd\u529b\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=\"#about-duchenne\">\u5173\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7<\/a><\/li><li><a href=\"#exon-51-skipping-therapies\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5<\/a><\/li><li><a href=\"#what-is-exon-51-skipping\">\u4ec0\u4e48\u662f\u5916\u663e\u5b50 51 \u8df3\u8dc3\uff1f<\/a><\/li><li><a href=\"#fda-approved-exon-51-skipping-therapies\">FDA \u83b7\u6279\u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5<\/a><ul><li><a href=\"#exondys-51-eteplirsen\">EXONDYS 51 (eteplirsen)<\/a><\/li><\/ul><\/li><li><a href=\"#which-mutations-are-amenable-to-exon-45-skipping\">\u9002\u5408\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684\u7f3a\u5931<\/a><\/li><li><a href=\"#clinical-trials-for-exon-51-skipping-therapies\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u7684\u4e34\u5e8a\u8bd5\u9a8c<\/a><ul><li><a href=\"#dyne-therapeutics-dyne-251-exon-51\">Dyne Therapeutics \u2013 DYNE-251<\/a><\/li><li><a href=\"#jiao-tong-university-le-051\">\u4ea4\u901a\u5927\u5b66 \u2013 LE051<\/a><\/li><li><a href=\"#bio-marin-pharmaceutical-bmn-351\">BioMarin \u5236\u836f \u2013 BMN 351<\/a><\/li><li><a href=\"#entrada-therapeutics\">Entrada Therapeutics \u2013 ENTR-601-51<\/a><\/li><li><a href=\"#nippon-shinyaku-ns-051-ncnp-04\">Nippon Shinyaku \u2013 NS-051\/NCNP-04<\/a><\/li><\/ul><\/li><li><a href=\"#conclusion\">\u7ed3\u8bba<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"about-duchenne\">\u5173\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u662f\u4e00\u79cd\u4e25\u91cd\u7684\u9057\u4f20\u6027\u75be\u75c5\uff0c\u4e3b\u8981\u5f71\u54cd\u7537\u5b69\uff0c\u5bfc\u81f4\u808c\u8089\u9010\u6e10\u65e0\u529b\u3001\u884c\u52a8\u4e0d\u4fbf\uff0c\u5e76\u6700\u7ec8\u5bfc\u81f4\u547c\u5438\u548c\u5fc3\u810f\u5e76\u53d1\u75c7\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u662f\u7531 DMD \u57fa\u56e0\u7a81\u53d8\u5f15\u8d77\u7684\uff0c\u8be5\u57fa\u56e0\u7f16\u7801\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u662f\u4e00\u79cd\u7a33\u5b9a\u808c\u8089\u7ec6\u80de\u819c\u7684\u5173\u952e\u86cb\u767d\u8d28\u3002\u5982\u679c\u6ca1\u6709\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u808c\u8089\u7ea4\u7ef4\u5728\u6536\u7f29\u8fc7\u7a0b\u4e2d\u66f4\u5bb9\u6613\u53d7\u635f\uff0c\u5bfc\u81f4\u808c\u8089\u9000\u5316\u548c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6807\u5fd7\u6027\u75c7\u72b6\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"exon-51-skipping-therapies\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD \u7684\u4e3b\u8981\u57fa\u56e0\u7a81\u53d8\u662f DMD \u57fa\u56e0\u4e2d\u7279\u5b9a\u5916\u663e\u5b50\u7684\u7f3a\u5931\uff0c\u4ece\u800c\u7834\u574f\u4e86\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u3002\u6cbb\u7597 DMD \u7684\u4e00\u79cd\u65b9\u6cd5\u662f\u5916\u663e\u5b50\u8df3\u8dc3\uff0c\u8fd9\u79cd\u6cbb\u7597\u7b56\u7565\u65e8\u5728\u7ed5\u8fc7\u57fa\u56e0\u7684\u7f3a\u9677\u90e8\u5206\uff0c\u4ece\u800c\u4ea7\u751f\u622a\u77ed\u4f46\u90e8\u5206\u529f\u80fd\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u5916\u663e\u5b50 51 \u8df3\u8dc3\u662f\u7814\u7a76\u6700\u591a\u7684\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u4e4b\u4e00\uff0c\u4e3a\u5927\u91cf DMD \u60a3\u8005\u5e26\u6765\u4e86\u5e0c\u671b\u3002\uff08<a href=\"https:\/\/dmdwarrior.com\/zh\/what-is-exon-skipping-and-how-does-it-work\/\" data-type=\"post\" data-id=\"1445\">\u4ec0\u4e48\u662f\u5916\u663e\u5b50\u8df3\u8dc3\uff1f<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-exon-51-skipping\">\u4ec0\u4e48\u662f\u5916\u663e\u5b50 51 \u8df3\u8dc3\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">DMD \u57fa\u56e0\u7531 79 \u4e2a\u5916\u663e\u5b50\u7ec4\u6210\uff0c\u8be5\u57fa\u56e0\u7a81\u53d8\u901a\u5e38\u6d89\u53ca\u7279\u5b9a\u5916\u663e\u5b50\u7684\u7f3a\u5931\u3002\u8fd9\u4e9b\u7f3a\u5931\u901a\u5e38\u4f1a\u5bfc\u81f4\u79fb\u7801\uff0c\u4ece\u800c\u963b\u6b62\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u3002\u5916\u663e\u5b50 51 \u8df3\u8dc3\u6d89\u53ca\u4f7f\u7528\u53cd\u4e49\u5be1\u6838\u82f7\u9178 (ASO) \u6765\u63a9\u76d6\u7a81\u53d8\u7684\u5916\u663e\u5b50\uff0c\u5e76\u5728 RNA \u526a\u63a5\u8fc7\u7a0b\u4e2d\u4fc3\u8fdb\u8be5\u5916\u663e\u5b50\u7684\u201c\u8df3\u8dc3\u201d\u3002\u901a\u8fc7\u8df3\u8fc7\u5916\u663e\u5b50 51\uff0c\u4e0b\u6e38\u5916\u663e\u5b50\u53ef\u4ee5\u8fde\u63a5\u5728\u4e00\u8d77\uff0c\u6062\u590d\u9605\u8bfb\u6846\u67b6\u5e76\u5141\u8bb8\u4ea7\u751f\u66f4\u77ed\u4f46\u529f\u80fd\u6027\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8be5\u7b56\u7565\u65e8\u5728\u6062\u590d\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u8be5\u86cb\u767d\u53ef\u4ee5\u5728\u4e00\u5b9a\u7a0b\u5ea6\u4e0a\u5f25\u8865\u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u7f3a\u4e4f\uff0c\u4ece\u800c\u6709\u53ef\u80fd\u51cf\u7f13\u75be\u75c5\u8fdb\u5c55\u5e76\u6539\u5584\u56e0\u5916\u663e\u5b50 51 \u8df3\u8dc3\u800c\u5bfc\u81f4\u7f3a\u5931\u7684\u60a3\u8005\u7684\u808c\u8089\u529f\u80fd\u3002\uff08\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/what-is-exon-deletion-in-duchenne-muscular-dystrophy-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD \u4e2d\u7684\u5916\u663e\u5b50\u7f3a\u5931\u662f\u4ec0\u4e48\uff1f<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"fda-approved-exon-51-skipping-therapies\">FDA \u83b7\u6279\u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"exondys-51-eteplirsen\">EXONDYS 51 (eteplirsen)<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">EXONDYS 51 (eteplirsen) \u662f\u9996\u4e2a\u83b7\u5f97 FDA \u6279\u51c6\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6cbb\u7597\u836f\u7269\uff0c\u9002\u7528\u4e8e\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u5df2\u786e\u8ba4\u53d1\u751f\u57fa\u56e0\u7a81\u53d8\u7684\u60a3\u8005\uff0c\u53ef\u901a\u8fc7\u8df3\u8fc7\u5916\u663e\u5b50 51 \u8fdb\u884c\u6cbb\u7597\u3002\u5bf9\u4e8e\u67d0\u4e9b\u60a3\u8005\uff0c\u5b83\u53ef\u4ee5\u5e2e\u52a9\u8eab\u4f53\u5236\u9020\u51fa\u8f83\u77ed\u5f62\u5f0f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\uff08\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/frequently-asked-questions-about-eteplirsen-exondys-51-for-dmd-treatment\/\" data-type=\"post\" data-id=\"2303\">Eteplirsen<\/a>)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">EXONDYS 51\uff08eteplirsen\uff09\u672a\u83b7\u5f97\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u7684\u6279\u51c6\u3002\uff08<a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/exondys\" target=\"_blank\" rel=\"noopener\">EMA<\/a>)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"which-mutations-are-amenable-to-exon-45-skipping\">\u9002\u5408\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684\u7f3a\u5931<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\u662f\u4e13\u95e8\u4e3a DMD \u57fa\u56e0\u5916\u663e\u5b50\u7f3a\u5931\u7684\u60a3\u8005\u8bbe\u8ba1\u7684\uff0c\u5982\u4e0b\u6240\u793a\u3002\u8fd9\u4e9b\u7f3a\u5931\u53ef\u4ee5\u901a\u8fc7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u6765\u89e3\u51b3\uff0c\u56e0\u4e3a\u8df3\u8fc7\u8fd9\u4e2a\u5916\u663e\u5b50\u53ef\u4ee5\u6062\u590d\u57fa\u56e0\u7684\u9605\u8bfb\u6846\u67b6\uff0c\u4ece\u800c\u4ea7\u751f\u4e00\u79cd\u7f29\u77ed\u4f46\u6709\u529f\u80fd\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"847\" height=\"1024\" src=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg\" alt=\"\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u53ef\u901a\u8fc7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u6cbb\u7597\u7684\u7a81\u53d8\u548c\u7f3a\u5931\" class=\"wp-image-4526\" title=\"\" srcset=\"https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-847x1024.jpg 847w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-248x300.jpg 248w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-768x929.jpg 768w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1270x1536.jpg 1270w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1693x2048.jpg 1693w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-10x12.jpg 10w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-347x420.jpg 347w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-150x181.jpg 150w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-300x363.jpg 300w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-696x842.jpg 696w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1068x1292.jpg 1068w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping-1920x2323.jpg 1920w, https:\/\/dmdwarrior.com\/wp-content\/uploads\/2025\/03\/amenable-to-exon-51-skipping.jpg 1984w\" sizes=\"auto, (max-width: 847px) 100vw, 847px\" \/><\/figure>\n\n\n\n<p class=\"wp-block-paragraph\">\u7814\u7a76\u8868\u660e\uff0c\u76f8\u5f53\u4e00\u90e8\u5206 DMD \u60a3\u8005\uff08\u7ea6 13-15%\uff09\u5b58\u5728\u53ef\u901a\u8fc7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u800c\u5b9e\u73b0\u7684\u7f3a\u5931\u3002\u8fd9\u4e9b\u7f3a\u5931\u901a\u5e38\u4f1a\u5f71\u54cd DMD \u57fa\u56e0\u7684\u5173\u952e\u90e8\u5206\uff0c\u5bfc\u81f4\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u529f\u80fd\u4e27\u5931\u3002\u901a\u8fc7\u8df3\u8dc3\u5916\u663e\u5b50 51\uff0c\u8fd9\u4e9b\u60a3\u8005\u53ef\u4ee5\u4ea7\u751f\u6539\u826f\u5f62\u5f0f\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u4ece\u800c\u6709\u53ef\u80fd\u4fdd\u6301\u808c\u8089\u5b8c\u6574\u6027\u3001\u51cf\u7f13\u75be\u75c5\u8fdb\u5c55\u5e76\u6539\u5584\u751f\u6d3b\u8d28\u91cf\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u503c\u5f97\u6ce8\u610f\u7684\u662f\uff0c\u5916\u663e\u5b50 51 \u8df3\u8dc3\u4ec5\u5bf9\u67d0\u4e9b\u57fa\u56e0\u7a81\u53d8\u6709\u6548\uff0c\u60a3\u8005\u5fc5\u987b\u63a5\u53d7\u57fa\u56e0\u68c0\u6d4b\u4ee5\u786e\u5b9a\u4ed6\u4eec\u662f\u5426\u9002\u5408\u63a5\u53d7\u5916\u663e\u5b50 51 \u7597\u6cd5\u3002\u4e0b\u4e00\u4ee3\u6d4b\u5e8f (NGS) \u548c\u57fa\u56e0\u6d4b\u5e8f\u6280\u672f\u4f7f\u8bc6\u522b\u8fd9\u4e9b\u7f3a\u5931\u5e76\u8bc4\u4f30\u5176\u5bf9\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u7684\u9002\u5e94\u6027\u53d8\u5f97\u66f4\u52a0\u5bb9\u6613\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"clinical-trials-for-exon-51-skipping-therapies\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u7684\u4e34\u5e8a\u8bd5\u9a8c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u867d\u7136\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u4ee3\u8868\u4e86 DMD \u6cbb\u7597\u7684\u91cd\u5927\u8fdb\u6b65\uff0c\u4f46\u4ecd\u5b58\u5728\u4e00\u4e9b\u6311\u6218\u3002\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u5728\u957f\u671f\u76ca\u5904\u548c\u529f\u80fd\u6539\u5584\u65b9\u9762\u7684\u6709\u6548\u6027\u4ecd\u5728\u7814\u7a76\u4e2d\u3002\u6b64\u5916\uff0c\u60a3\u8005\u53cd\u5e94\u7684\u53ef\u53d8\u6027\uff08\u5305\u62ec\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\u7a0b\u5ea6\u53ca\u5176\u5982\u4f55\u8f6c\u5316\u4e3a\u529f\u80fd\u6539\u5584\uff09\u5e26\u6765\u4e86\u6301\u7eed\u7684\u6311\u6218\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4ee5\u4e0b\u662f\u6709\u5e0c\u671b\u7684\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7814\u7a76\uff1a<\/strong><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"dyne-therapeutics-dyne-251-exon-51\">Dyne Therapeutics \u2013 DYNE-251<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">DYNE-251 \u662f\u4e00\u79cd\u7814\u7a76\u6027\u6cbb\u7597\u836f\u7269\uff0c\u76ee\u524d\u6b63\u5728\u8fdb\u884c 1\/2 \u671f\u5168\u7403 DELIVER \u4e34\u5e8a\u8bd5\u9a8c\uff0c\u8be5\u8bd5\u9a8c\u9488\u5bf9\u7684\u662f\u60a3\u6709 DMD \u5e76\u63a5\u53d7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u6cbb\u7597\u7684\u60a3\u8005\u3002\uff08\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/dyne-therapeutics-announces-phase-1-2-deliver-trial-of-dyne-251-in-duchenne-muscular-dystrophy\/\">DYNE-251<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"jiao-tong-university-le-051\">\u4ea4\u901a\u5927\u5b66 \u2013 LE051<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e0a\u6d77\u4ea4\u901a\u5927\u5b66\u533b\u5b66\u9662\u5ba3\u5e03\u5f00\u5c55\u4e00\u9879\u4e34\u5e8a\u8bd5\u9a8c\uff0c\u65e8\u5728\u8bc4\u4f30 LE051 \u5355\u6b21\u9759\u8109\u8f93\u6ce8\u5bf9\u63a5\u53d7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u7684 DMD \u60a3\u8005\u7684\u5b89\u5168\u6027\u3001\u8010\u53d7\u6027\u548c\u6709\u6548\u6027\u3002\uff08\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/shanghai-jiao-tong-university-school-of-medicine-begins-clinical-trials-of-le051-exon-51-skipping-therapy\/\">LE051<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"bio-marin-pharmaceutical-bmn-351\">BioMarin \u5236\u836f \u2013 BMN 351<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">BMN 351 \u662f\u9488\u5bf9\u60a3\u6709\u5916\u663e\u5b50 51 \u6613\u611f DMD \u7684\u7537\u5b69\u7684\u65b0\u4e00\u4ee3\u53cd\u4e49\u5be1\u6838\u82f7\u9178 (ASO) \u7597\u6cd5\u3002ASO \u662f\u5f71\u54cd\u7ec6\u80de\u5236\u9020\u67d0\u4e9b\u86cb\u767d\u8d28\u7684\u836f\u7269\u3002\u5b83\u4eec\u88ab\u7528\u4e8e\u6cbb\u7597\u591a\u79cd\u4e0d\u540c\u7c7b\u578b\u7684\u9057\u4f20\u5065\u5eb7\u72b6\u51b5\u3002BMN 351 \u662f\u4e00\u79cd\u7814\u7a76\u836f\u7269\uff0c\u8fd9\u610f\u5473\u7740\u5b83\u5c1a\u672a\u88ab\u53d1\u73b0\u5bf9\u4eba\u4f53\u5b89\u5168\u6709\u6548\uff0c\u4e5f\u672a\u83b7\u51c6\u5728\u4e34\u5e8a\u8bd5\u9a8c\u4e4b\u5916\u4f7f\u7528\u3002\uff08\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/clinicaltrials.biomarin.com\/clinical-trial\/a-phase-1-2-study-to-assess-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-bmn-351-in-participants-with-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noopener\">BMN351<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"entrada-therapeutics\">Entrada Therapeutics \u2013 ENTR-601-51<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">ENTR-601-51 \u662f\u4e00\u79cd\u7814\u7a76\u6027\u7597\u6cd5\uff0c\u7528\u4e8e\u6cbb\u7597\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u8005\uff0c\u76ee\u524d\u6b63\u5728\u8bc4\u4f30\u5176\u6062\u590d mRNA \u9605\u8bfb\u6846\u67b6\u548c\u5141\u8bb8\u7ffb\u8bd1\u7565\u5fae\u7f29\u77ed\u4f46\u4ecd\u6709\u529f\u80fd\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u6f5c\u529b\u3002\uff08\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/www.entradatx.com\/pipeline\" target=\"_blank\" rel=\"noopener\">ENTR-601-51<\/a>)<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"nippon-shinyaku-ns-051-ncnp-04\">Nippon Shinyaku \u2013 NS-051\/NCNP-04<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">DMD \u662f\u4e00\u79cd\u7531\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7f3a\u4e4f\u5f15\u8d77\u7684\u8fdb\u884c\u6027\u808c\u8089\u840e\u7f29\u75c7\u3002\u5b83\u4f1a\u5bfc\u81f4\u9aa8\u9abc\u808c\u3001\u5fc3\u808c\u548c\u547c\u5438\u808c\u65e0\u529b\u3002\u591a\u79cd\u7c7b\u578b\u7684\u57fa\u56e0\u7a81\u53d8\u53ef\u5bfc\u81f4 DMD\uff0cNS-051\/NCNP-04 \u6b63\u5728\u5f00\u53d1\u4e2d\uff0c\u7528\u4e8e\u6cbb\u7597\u5df2\u786e\u8bca\u57fa\u56e0\u7a81\u53d8\u4e14\u9002\u5408\u63a5\u53d7\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u7684\u60a3\u8005\u3002\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/fda-grants-orphan-drug-designation-to-ns-051-ncnp-04-for-the-treatment-of-duchenne-muscular-dystrophy\/\">FDA \u6388\u4e88 NS-051\/NCNP-04 \u5b64\u513f\u836f\u8d44\u683c\u8ba4\u5b9a<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"conclusion\">\u7ed3\u8bba<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u4ee3\u8868\u4e86\u4e00\u79cd\u6709\u524d\u9014\u7684\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6cbb\u7597\u65b9\u6cd5\uff0c\u4e3a\u60a3\u6709\u7279\u5b9a\u7a81\u53d8\u4e14\u9002\u5408\u8be5\u6280\u672f\u7684\u60a3\u8005\u5e26\u6765\u4e86\u5e0c\u671b\u3002\u867d\u7136\u957f\u671f\u529f\u80fd\u76ca\u5904\u4ecd\u5728\u7814\u7a76\u4e2d\uff0c\u4f46\u5916\u663e\u5b50 51 \u8df3\u8dc3\u7597\u6cd5\u6807\u5fd7\u7740\u5bf9\u6297\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e00\u5927\u8fdb\u6b65\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6301\u7eed\u7684\u7814\u7a76\u3001\u957f\u671f\u7684\u4e34\u5e8a\u8bd5\u9a8c\u4ee5\u53ca\u57fa\u56e0\u68c0\u6d4b\u548c\u4e2a\u6027\u5316\u533b\u7597\u7684\u8fdb\u4e00\u6b65\u53d1\u5c55\u5bf9\u4e8e\u4f18\u5316\u8fd9\u4e9b\u7597\u6cd5\u5e76\u5728\u672a\u6765\u5c06\u5176\u5e94\u7528\u6269\u5c55\u5230\u66f4\u591a\u7684 DMD \u60a3\u8005\u81f3\u5173\u91cd\u8981\u3002<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u9605\u8bfb\u66f4\u591a<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/zh\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e34\u5e8a\u8bd5\u9a8c\uff08\u6240\u6709\u7814\u7a76\u5217\u8868\uff09<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Duchenne Exon 51 skipping therapies are innovative treatments aimed at addressing the genetic mutation responsible for Duchenne muscular dystrophy (DMD). By skipping exon 51 in the dystrophin gene, these therapies help produce a shorter, functional version of the dystrophin protein, potentially slowing disease progression and improving quality of life for patients. This cutting-edge approach offers [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3444,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[122,193,194,77,178],"class_list":["post-3429","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-exon-51","tag-exon-51-skipping","tag-exon-51-treatment","tag-exon-skipping","tag-exon-skipping-therapies"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3429","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=3429"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/3429\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/3444"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=3429"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=3429"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=3429"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}