{"id":2418,"date":"2025-01-14T11:43:29","date_gmt":"2025-01-14T08:43:29","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=2418"},"modified":"2025-03-07T14:58:15","modified_gmt":"2025-03-07T11:58:15","slug":"wave-life-sciences-announces-plans-for-exon-53-skipping-study-in-2025","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/wave-life-sciences-announces-plans-for-exon-53-skipping-study-in-2025\/","title":{"rendered":"Wave Life Sciences \u5ba3\u5e03\u8ba1\u5212\u4e8e 2025 \u5e74\u5f00\u5c55\u5916\u663e\u5b50 53 \u8df3\u8dc3\u7814\u7a76"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Alpha-1 \u6297\u80f0\u86cb\u767d\u9176\u7f3a\u4e4f\u75c7 (AATD)\u3001\u4ea8\u5ef7\u987f\u6c0f\u75c5 (HD)\u3001\u80a5\u80d6\u75c7\u548c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u662f\u8be5\u516c\u53f8\u5bfb\u6c42\u89e3\u51b3\u7684\u4e3b\u8981\u672a\u6ee1\u8db3\u533b\u7597\u9700\u6c42\u3002\u8be5\u6d88\u606f\u662f\u5728\u8be5\u516c\u53f8\u4e8e 2025 \u5e74 1 \u6708 13 \u65e5\u5728\u7b2c 43 \u5c4a\u6469\u6839\u5927\u901a\u5e74\u5ea6\u533b\u7597\u4fdd\u5065\u4f1a\u8bae\u4e0a\u53d1\u8868\u6f14\u8bb2\u4e4b\u524d\u53d1\u5e03\u7684\u3002<\/p>\n\n\n\n<h2 class=\"wp-block-heading\">\u5916\u663e\u5b50 53 \u8df3\u8dc3\u7814\u7a76\u8ba1\u5212<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>WVE-N531 \u662f\u4e00\u79cd\u5916\u663e\u5b50\u8df3\u8dc3\u5be1\u6838\u82f7\u9178\uff0c\u65e8\u5728\u8bf1\u5bfc\u5185\u6e90\u6027\u3001\u529f\u80fd\u6027\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u751f\uff0c\u7528\u4e8e\u6cbb\u7597\u9002\u5408\u8fdb\u884c\u5916\u663e\u5b50 53 \u8df3\u8dc3\u7684 DMD \u7537\u5b69\u3002<\/li>\n\n\n\n<li>2024 \u5e74\u7684\u4e3b\u8981\u6210\u5c31\n<ul class=\"wp-block-list\">\n<li>WVE-N531 \u7b2c 2 \u9636\u6bb5 FORWARD-53 \u7814\u7a76\u7684\u4e2d\u671f\u7ed3\u679c\u663e\u793a\uff0c\u9ad8\u5ea6\u4e00\u81f4\uff0c\u5e73\u5747\u808c\u8089\u542b\u91cf\u8c03\u6574\u540e\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u4e3a 9.0%\uff08\u8303\u56f4\uff1a4.6-13.9%\uff09\uff0c\u4e00\u6d41\u7684\u808c\u8089\u8f93\u9001\uff0c\u6539\u5584\u808c\u8089\u5065\u5eb7\u7684\u591a\u9879\u6307\u6807\uff0c\u4ee5\u53ca\u5b89\u5168\u548c\u826f\u597d\u7684\u8010\u53d7\u6027\u3002<\/li>\n<\/ul>\n<\/li>\n\n\n\n<li>\u9884\u671f\u5373\u5c06\u5230\u6765\u7684\u91cc\u7a0b\u7891\uff1a\n<ul class=\"wp-block-list\">\n<li>FORWARD-53 \u8bd5\u9a8c\u6b63\u5728\u8fdb\u884c\u4e2d\uff0c\u6240\u6709\u60a3\u8005\u5747\u9009\u62e9\u5728\u7814\u7a76\u8ba1\u5212\u7684\u5ef6\u957f\u90e8\u5206\u4e2d\u7ee7\u7eed\u63a5\u53d7\u6bcf\u6708\u670d\u7528 WVE-N531 \u7684\u6cbb\u7597\u3002<\/li>\n\n\n\n<li>Wave \u9884\u8ba1\u5c06\u5728 2025 \u5e74\u7b2c\u4e00\u5b63\u5ea6\u63d0\u4f9b 48 \u5468\u7684 FORWARD-53 \u6570\u636e\uff0c\u5e76\u6536\u5230\u76d1\u7ba1\u673a\u6784\u5173\u4e8e\u52a0\u901f\u5ba1\u6279\u9014\u5f84\u7684\u53cd\u9988\u3002<\/li>\n<\/ul>\n<\/li>\n<\/ul>","protected":false},"excerpt":{"rendered":"<p>Alpha-1 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