{"id":7667,"date":"2026-04-22T19:42:28","date_gmt":"2026-04-22T16:42:28","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=7667"},"modified":"2026-04-22T19:42:30","modified_gmt":"2026-04-22T16:42:30","slug":"ck-levels-after-duchenne-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/ck-levels-after-duchenne-gene-therapy\/","title":{"rendered":"\u4e3a\u4ec0\u4e48\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u4e2d\uff0cCK\u3001AST \u548c ALT \u6570\u636e\u6bd4\u89c6\u9891\u66f4\u91cd\u8981"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff08DMD\uff09\u57fa\u56e0\u6cbb\u7597\u9886\u57df\u4e0d\u65ad\u53d1\u5c55\u7684\u8fdb\u7a0b\u4e2d\uff0c\u4e00\u4e2a\u95ee\u9898\u6b63\u53d8\u5f97\u8d8a\u6765\u8d8a\u6e05\u6670\uff1a <strong>\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u7684\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73\u8fdc\u6bd4\u7f51\u4e0a\u6d41\u4f20\u7684\u75c5\u6bd2\u89c6\u9891\u91cd\u8981\u5f97\u591a\u3002<\/strong>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bb6\u5c5e\u4eec\u6b63\u5728\u89c2\u770b\u4e00\u4e9b\u60c5\u611f\u51b2\u51fb\u529b\u5f88\u5f3a\u7684\u5ef6\u65f6\u89c6\u9891\uff0c\u8fd9\u4e9b\u89c6\u9891\u4f3c\u4e4e\u663e\u793a\u6cbb\u7597\u540e\u75c5\u60c5\u6709\u6240\u597d\u8f6c\u3002. <strong>\u7136\u800c\uff0c\u8fd9\u4e9b\u89c6\u89c9\u53d9\u8ff0\u901a\u5e38\u6ca1\u6709\u4f34\u968f\u751f\u5316\u6570\u636e\u2014\u2014\u7279\u522b\u662f\u808c\u9178\u6fc0\u9176 (CK)\u3001AST \u548c ALT \u6c34\u5e73\uff0c\u800c\u8fd9\u4e9b\u5bf9\u4e8e\u8bc4\u4f30\u7597\u6548\u548c\u5b89\u5168\u6027\u81f3\u5173\u91cd\u8981\u3002.<\/strong> <\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u8fd9\u5c31\u5f15\u51fa\u4e86\u4e00\u4e2a\u5173\u952e\u95ee\u9898\uff1a<br><strong>\u4e3a\u4ec0\u4e48\u6211\u4eec\u770b\u5230\u7684\u662f\u89c6\u9891\uff0c\u800c\u4e0d\u662f\u5e94\u8be5\u7528\u6765\u5b9a\u4e49\u6cbb\u7597\u7ed3\u679c\u7684\u751f\u7269\u6807\u5fd7\u7269\u6570\u636e\uff1f<\/strong><\/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=\"#the-problem-viral-videos-are-replacing-clinical-evidence\">\u95ee\u9898\uff1a\u75c5\u6bd2\u5f0f\u4f20\u64ad\u7684\u89c6\u9891\u6b63\u5728\u53d6\u4ee3\u4e34\u5e8a\u8bc1\u636e<\/a><\/li><li><a href=\"#ck-levels-after-duchenne-gene-therapy-the-core-biomarker\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e CK \u6c34\u5e73\uff1a\u6838\u5fc3\u751f\u7269\u6807\u5fd7\u7269<\/a><\/li><li><a href=\"#ast-and-alt-essential-safety-and-muscle-biomarkers\">AST \u548c ALT\uff1a\u91cd\u8981\u7684\u5b89\u5168\u6027\u548c\u808c\u8089\u751f\u7269\u6807\u5fd7\u7269<\/a><\/li><li><a href=\"#why-are-ck-levels-after-duchenne-gene-therapy-not-fully-disclosed\">\u4e3a\u4ec0\u4e48\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u7684CK\u6c34\u5e73\u6ca1\u6709\u5b8c\u5168\u516c\u5f00\uff1f<\/a><\/li><li><a href=\"#the-ethical-issue-emotion-vs-evidence\">\u4f26\u7406\u95ee\u9898\uff1a\u60c5\u611f\u4e0e\u8bc1\u636e<\/a><\/li><li><a href=\"#what-real-transparency-should-look-like\">\u771f\u6b63\u7684\u900f\u660e\u5ea6\u5e94\u8be5\u662f\u4ec0\u4e48\u6837\u7684<\/a><\/li><li><a href=\"#the-consequences-of-continued-opacity\">\u6301\u7eed\u4e0d\u900f\u660e\u7684\u540e\u679c<\/a><\/li><li><a href=\"#frequently-asked-questions-faq\">\u5e38\u89c1\u95ee\u9898\u89e3\u7b54 (FAQ)<\/a><\/li><li><a href=\"#final-thoughts\">\u6700\u540e\u7684\u60f3\u6cd5<\/a><\/li><li><a href=\"#academic-sources-and-references\">\u5b66\u672f\u8d44\u6e90\u548c\u53c2\u8003\u6587\u732e<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-problem-viral-videos-are-replacing-clinical-evidence\">\u95ee\u9898\uff1a\u75c5\u6bd2\u5f0f\u4f20\u64ad\u7684\u89c6\u9891\u6b63\u5728\u53d6\u4ee3\u4e34\u5e8a\u8bc1\u636e<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-rise-of-anecdotal-evidence-in-dmd\">DMD\u4e2d\u8f76\u4e8b\u8bc1\u636e\u7684\u5174\u8d77<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u57fa\u56e0\u6cbb\u7597\u540e\uff0c\u513f\u7ae5\u80fd\u591f\u884c\u8d70\u3001\u722c\u697c\u68af\u6216\u72ec\u7acb\u7ad9\u7acb\u7684\u89c6\u9891\u88ab\u5e7f\u6cdb\u4f20\u64ad\u3002\u867d\u7136\u8fd9\u4e9b\u753b\u9762\u53ef\u80fd\u771f\u5b9e\uff0c\u4f46\u5b83\u4eec\u5e76\u4e0d\u80fd\u4f5c\u4e3a\u6cbb\u7597\u6210\u529f\u7684\u79d1\u5b66\u53ef\u9760\u6307\u6807\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5b83\u4eec\u7f3a\u4e4f\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u53d7\u63a7\u6761\u4ef6<\/li>\n\n\n\n<li>\u6807\u51c6\u5316\u529f\u80fd\u8bc4\u5206<\/li>\n\n\n\n<li>\u57fa\u7ebf\u6bd4\u8f83<\/li>\n\n\n\n<li>\u957f\u671f\u968f\u8bbf<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6700\u91cd\u8981\u7684\u662f\uff0c\u4ed6\u4eec\u7f3a\u4e4f\u786e\u5b9a\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u808c\u8089\u635f\u4f24\u662f\u5426\u771f\u6b63\u51cf\u5c11\u6240\u9700\u7684 CK \u6c34\u5e73\uff0c\u4ee5\u53ca\u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u7684\u6570\u636e\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e86\u89e3\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/what-is-creatine-kinase-ck\/\" target=\"_blank\" data-type=\"post\" data-id=\"2167\" rel=\"noreferrer noopener\">\u4ec0\u4e48\u662f\u808c\u9178\u6fc0\u9176\uff08CK\uff09\uff1f<\/a><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-this-is-dangerous\">\u4e3a\u4ec0\u4e48\u8fd9\u6837\u505a\u5f88\u5371\u9669<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f53\u8f76\u4e8b\u5185\u5bb9\u53d6\u4ee3\u5ba2\u89c2\u6570\u636e\u65f6\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5bb6\u5ead\u53ef\u80fd\u4f1a\u4ea7\u751f\u4e0d\u5207\u5b9e\u9645\u7684\u671f\u671b\u3002<\/li>\n\n\n\n<li>\u6cbb\u7597\u51b3\u7b56\u53ef\u80fd\u53d7\u5230\u4fe1\u606f\u4e0d\u5b8c\u6574\u7684\u5f71\u54cd\u3002<\/li>\n\n\n\n<li>\u79d1\u5b66\u4e25\u8c28\u6027\u53d7\u5230\u635f\u5bb3<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u4e0d\u662f\u6c9f\u901a\u95ee\u9898\uff0c\u800c\u662f\u6570\u636e\u900f\u660e\u5ea6\u95ee\u9898\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"ck-levels-after-duchenne-gene-therapy-the-core-biomarker\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e CK \u6c34\u5e73\uff1a\u6838\u5fc3\u751f\u7269\u6807\u5fd7\u7269<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"what-ck-levels-represent\">CK\u6c34\u5e73\u4ee3\u8868\u4ec0\u4e48<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u808c\u9178\u6fc0\u9176 (CK) \u662f\u4e00\u79cd\u5173\u952e\u9176\uff0c\u5f53\u808c\u8089\u7ea4\u7ef4\u53d7\u635f\u65f6\u4f1a\u91ca\u653e\u51fa\u6765\u3002\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e2d\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73\u901a\u5e38\u6781\u9ad8\u3002<\/strong><\/li>\n\n\n\n<li>\u6301\u7eed\u5347\u9ad8\u53cd\u6620\u51fa\u808c\u8089\u6301\u7eed\u9000\u5316\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u57fa\u56e0\u6cbb\u7597\u540e\uff0c\u5e94\u5bc6\u5207\u76d1\u6d4b CK \u6c34\u5e73\uff0c\u4ee5\u786e\u5b9a\u662f\u5426\uff1a<\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u808c\u8089\u635f\u4f24\u6b63\u5728\u51cf\u5c11<\/li>\n\n\n\n<li>\u8be5\u7597\u6cd5\u6b63\u5728\u4ea7\u751f\u751f\u7269\u6548\u5e94\u3002<\/li>\n\n\n\n<li>\u75be\u75c5\u8fdb\u7a0b\u6b63\u5728\u53d1\u751f\u6539\u53d8<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-ck-data-must-be-public\">\u4e3a\u4ec0\u4e48CK\u6570\u636e\u5fc5\u987b\u516c\u5f00<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u65e0\u6cd5\u68c0\u6d4b\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6539\u5584\u7684\u8bf4\u6cd5\u5c1a\u672a\u5f97\u5230\u8bc1\u5b9e\u3002<\/li>\n\n\n\n<li>\u529f\u80fd\u6027\u6539\u5584\u4e0e\u751f\u7269\u5b66\u53d8\u5316\u65e0\u5173<\/li>\n\n\n\n<li>\u4e34\u5e8a\u533b\u751f\u548c\u5bb6\u5c5e\u65e0\u6cd5\u505a\u51fa\u77e5\u60c5\u51b3\u5b9a<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>CK \u4e0d\u662f\u53ef\u6709\u53ef\u65e0\u7684\uff0c\u800c\u662f\u5fc5\u4e0d\u53ef\u5c11\u7684\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"ast-and-alt-essential-safety-and-muscle-biomarkers\">AST \u548c ALT\uff1a\u91cd\u8981\u7684\u5b89\u5168\u6027\u548c\u808c\u8089\u751f\u7269\u6807\u5fd7\u7269<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"beyond-liver-function\">\u8d85\u8d8a\u809d\u529f\u80fd<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">AST\uff08\u5929\u51ac\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff09\u548c ALT\uff08\u4e19\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff09\u5e38\u88ab\u8bef\u89e3\u4e3a\u7eaf\u7cb9\u7684\u809d\u9176\u3002. <strong>\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0c\u5b83\u4eec\u4e5f\u53cd\u6620\u4e86\u808c\u8089\u7684\u5b8c\u6574\u6027\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u57fa\u56e0\u6cbb\u7597\u540e\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>AST\/ALT\u5347\u9ad8\u53ef\u80fd\u63d0\u793a\u808c\u8089\u5206\u89e3\u6216\u6cbb\u7597\u76f8\u5173\u6bd2\u6027\u3002<\/strong><\/li>\n\n\n\n<li>\u8fd9\u4e9b\u6307\u6807\u7684\u53d8\u5316\u63d0\u4f9b\u4e86\u5173\u952e\u7684\u5b89\u5168\u4fe1\u53f7\u3002<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-transparency-gap\">\u900f\u660e\u5ea6\u5dee\u8ddd<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c3d\u7ba1\u5b83\u4eec\u5f88\u91cd\u8981\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>AST \u548c ALT \u6570\u636e\u5e76\u672a\u59cb\u7ec8\u5982\u4e00\u5730\u516c\u5f00\u62ab\u9732\u3002<\/li>\n\n\n\n<li>\u516c\u5f00\u62a5\u9053\u5f80\u5f80\u4e0d\u5b8c\u6574\u6216\u6709\u5ef6\u8fdf\u3002<\/li>\n\n\n\n<li>\u5bb6\u5ead\u65e0\u6cd5\u83b7\u5f97\u5b8c\u6574\u7684\u5b89\u5168\u4fdd\u969c\u4fe1\u606f\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u9ad8\u98ce\u9669\u7597\u6cd5\u4e2d\uff0c\u8fd9\u79cd\u7f3a\u4e4f\u900f\u660e\u5ea6\u662f\u4e0d\u53ef\u63a5\u53d7\u7684\u3002.<\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"why-are-ck-levels-after-duchenne-gene-therapy-not-fully-disclosed\">\u4e3a\u4ec0\u4e48\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u7684CK\u6c34\u5e73\u6ca1\u6709\u5b8c\u5168\u516c\u5f00\uff1f<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-accountability-problem\">\u95ee\u8d23\u95ee\u9898<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u7814\u53d1\u57fa\u56e0\u7597\u6cd5\u7684\u5236\u836f\u516c\u53f8\u9762\u4e34\u7740\u6fc0\u70c8\u7684\u7ade\u4e89\u73af\u5883\u3002\u7136\u800c\uff0c\u8fd9\u5e76\u4e0d\u80fd\u6210\u4e3a\u9690\u7792\u5173\u952e\u751f\u7269\u6807\u5fd7\u7269\u6570\u636e\u7684\u7406\u7531\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f53\u516c\u53f8\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7a81\u51fa\u89c6\u89c9\u6539\u8fdb<\/li>\n\n\n\n<li>\u5206\u4eab\u90e8\u5206\u7ed3\u679c<\/li>\n\n\n\n<li>\u5ef6\u8fdf\u62ab\u9732\u751f\u7269\u6807\u5fd7\u7269<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5b83\u4eec\u9020\u6210\u4fe1\u606f\u4e0d\u5bf9\u79f0\uff0c\u4f7f\u60a3\u8005\u548c\u4e34\u5e8a\u533b\u751f\u5904\u4e8e\u4e0d\u5229\u5730\u4f4d\u3002.<\/strong><\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"the-role-of-regulators\">\u76d1\u7ba1\u673a\u6784\u7684\u4f5c\u7528<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u7b49\u673a\u6784\u8d1f\u8d23\u786e\u4fdd\u5b89\u5168\u6027\u548c\u900f\u660e\u5ea6\u3002\u7136\u800c\uff0c\u516c\u4f17\u83b7\u53d6\u4ee5\u4e0b\u4fe1\u606f\u7684\u6743\u5229\u6709\u9650\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>CK\u8d8b\u52bf<\/li>\n\n\n\n<li>AST\/ALT \u6570\u636e<\/li>\n\n\n\n<li>\u957f\u671f\u7ed3\u679c<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u8bb8\u591a\u60c5\u51b5\u4e0b\u4ecd\u7136\u6709\u9650\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u76d1\u7ba1\u76d1\u7763\u5fc5\u987b\u4e0e\u65f6\u4ff1\u8fdb\uff0c\u4f18\u5148\u8003\u8651\u5b9e\u65f6\u6570\u636e\u900f\u660e\u5ea6\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-ethical-issue-emotion-vs-evidence\">\u4f26\u7406\u95ee\u9898\uff1a\u60c5\u611f\u4e0e\u8bc1\u636e<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5bb6\u5ead\u6210\u5458\u5206\u4eab\u89c6\u9891\u5e76\u975e\u95ee\u9898\u7684\u6839\u6e90\u2014\u2014\u4ed6\u4eec\u53ea\u662f\u5728\u5e94\u5bf9\u4fe1\u606f\u83b7\u53d6\u7684\u532e\u4e4f\u3002\u7136\u800c\uff0c\u5f53\u8fd9\u4e9b\u89c6\u9891\u4e3b\u5bfc\u4e86\u53d9\u8ff0\u65b9\u5f0f\u65f6\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u4ed6\u4eec\u53ef\u80fd\u4f1a\u65e0\u610f\u4e2d\u8bef\u5bfc\u4ed6\u4eba\u3002<\/li>\n\n\n\n<li>\u4ed6\u4eec\u628a\u6ce8\u610f\u529b\u4ece\u53ef\u8861\u91cf\u7684\u7ed3\u679c\u4e0a\u8f6c\u79fb\u5f00\u6765\u3002<\/li>\n\n\n\n<li>\u5b83\u4eec\u7ed9\u60a3\u8005\u7fa4\u4f53\u5e26\u6765\u4e86\u538b\u529b\u3002<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u771f\u6b63\u7684\u95ee\u9898\u5728\u4e8e\u8fd9\u6837\u4e00\u4e2a\u7cfb\u7edf\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6570\u636e\u532e\u4e4f\u3002<\/li>\n\n\n\n<li>\u60c5\u611f\u586b\u8865\u4e86\u7a7a\u767d<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u8fd9\u79cd\u4e0d\u5e73\u8861\u5fc5\u987b\u5f97\u5230\u7ea0\u6b63\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-real-transparency-should-look-like\">\u771f\u6b63\u7684\u900f\u660e\u5ea6\u5e94\u8be5\u662f\u4ec0\u4e48\u6837\u7684<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u4e3a\u4e86\u6062\u590d\u4fe1\u4efb\u548c\u79d1\u5b66\u8bda\u4fe1\uff0c\u8be5\u9886\u57df\u5fc5\u987b\u91c7\u7528\u65b0\u7684\u6807\u51c6\uff1a<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"1-routine-disclosure-of-ck-levels-after-duchenne-gene-therapy\">1. \u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u5e38\u89c4\u62ab\u9732\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u57fa\u7ebf\u4e0e\u6cbb\u7597\u540e\u6bd4\u8f83<\/li>\n\n\n\n<li>\u7eb5\u5411\u8ffd\u8e2a\uff08\u6708\/\u5e74\uff09<\/li>\n\n\n\n<li>\u5bf9\u8d8b\u52bf\u7684\u6e05\u6670\u89e3\u8bfb<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"2-full-reporting-of-ast-and-alt\">2. \u5168\u9762\u62a5\u544aAST\u548cALT\u503c\u3002<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5b89\u5168\u76d1\u63a7<\/li>\n\n\n\n<li>\u4e0d\u826f\u4e8b\u4ef6\u68c0\u6d4b<\/li>\n\n\n\n<li>\u8bed\u5883\u89e3\u91ca<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"3-public-access-to-data\">3. \u6570\u636e\u516c\u5f00<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5f00\u653e\u6ce8\u518c<\/li>\n\n\n\n<li>\u53ef\u4e0b\u8f7d\u6570\u636e\u96c6<\/li>\n\n\n\n<li>\u53ef\u89c6\u5316\u4eea\u8868\u76d8<\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"4-independent-validation\">4. \u72ec\u7acb\u9a8c\u8bc1<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u7b2c\u4e09\u65b9\u5206\u6790<\/li>\n\n\n\n<li>\u5b66\u672f\u5408\u4f5c<\/li>\n\n\n\n<li>\u76d1\u7ba1\u5ba1\u67e5<\/li>\n<\/ul>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-consequences-of-continued-opacity\">\u6301\u7eed\u4e0d\u900f\u660e\u7684\u540e\u679c<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5982\u679c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u7684 CK \u6c34\u5e73\u4ecd\u672a\u516c\u5f00\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u4eba\u4eec\u5bf9\u57fa\u56e0\u7597\u6cd5\u7684\u4fe1\u4efb\u5ea6\u5c06\u4f1a\u4e0b\u964d<\/li>\n\n\n\n<li>\u5bb6\u5ead\u53ef\u80fd\u4f1a\u505a\u51fa\u9519\u8bef\u7684\u51b3\u5b9a<\/li>\n\n\n\n<li>\u79d1\u5b66\u8fdb\u6b65\u5c06\u4f1a\u653e\u7f13<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u900f\u660e\u5ea6\u4e0d\u4ec5\u662f\u79d1\u5b66\u8981\u6c42\uff0c\u66f4\u662f\u9053\u5fb7\u4e49\u52a1\u3002.<\/strong><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"frequently-asked-questions-faq\">\u5e38\u89c1\u95ee\u9898\u89e3\u7b54 (FAQ)<\/h2>\n\n\n<div id=\"rank-math-faq\" class=\"rank-math-block\">\n<div class=\"rank-math-list\">\n<div id=\"faq-question-1776705232498\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u4ec0\u4e48\u662f CK\uff1f\u5b83\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u4e2d\u4e3a\u4f55\u5982\u6b64\u91cd\u8981\uff1f<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u808c\u9178\u6fc0\u9176 (CK) \u662f\u4e00\u79cd\u5728\u808c\u8089\u7ea4\u7ef4\u53d7\u635f\u65f6\u91ca\u653e\u5230\u8840\u6db2\u4e2d\u7684\u9176\u3002\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0c\u7531\u4e8e\u808c\u8089\u6301\u7eed\u5206\u89e3\uff0cCK \u6c34\u5e73\u901a\u5e38\u975e\u5e38\u9ad8\u3002.<\/p>\n<p>\u57fa\u56e0\u6cbb\u7597\u540e\uff0cCK\uff08\u808c\u9178\u6fc0\u9176\uff09\u975e\u5e38\u91cd\u8981\uff0c\u56e0\u4e3a\uff1a<\/p>\n<p>\u2022 CK \u6c34\u5e73\u964d\u4f4e\u53ef\u80fd\u8868\u660e\u808c\u8089\u635f\u4f24\u51cf\u8f7b\u3002<br \/>\u2022 CK \u6c34\u5e73\u7a33\u5b9a\u6216\u5347\u9ad8\u53ef\u80fd\u63d0\u793a\u75be\u75c5\u6d3b\u52a8\u6301\u7eed\u5b58\u5728<br \/>\u2022 CK \u8d8b\u52bf\u6709\u52a9\u4e8e\u8bc4\u4f30\u6cbb\u7597\u662f\u5426\u5177\u6709\u751f\u7269\u5b66\u6548\u5e94\uff0c\u800c\u4e0d\u4ec5\u4ec5\u662f\u53ef\u89c1\u6548\u5e94\u3002<\/p>\n<p>\u7b80\u800c\u8a00\u4e4b\uff1a <strong>CK \u63d0\u4f9b\u4e86\u5ba2\u89c2\u7684\u751f\u5316\u8bc1\u636e\uff0c\u8fd9\u662f\u5355\u51ed\u89c6\u9891\u65e0\u6cd5\u63d0\u4f9b\u7684\u3002.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705321179\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u57fa\u56e0\u6cbb\u7597\u540e\u7684\u6539\u5584\u89c6\u9891\u80fd\u4f5c\u4e3a\u53ef\u9760\u7684\u8bc1\u636e\u5417\uff1f<\/h3>\n<div class=\"rank-math-answer\">\n\n<p><strong>\u4e0d\u2014\u2014\u89c6\u9891\u4e0d\u80fd\u4f5c\u4e3a\u53ef\u9760\u7684\u79d1\u5b66\u8bc1\u636e\u3002.<\/strong><\/p>\n<p>\u867d\u7136\u5b83\u4eec\u5c55\u73b0\u4e86\u771f\u5b9e\u7684\u77ac\u95f4\uff0c\u4f46\u5b83\u4eec\u7f3a\u4e4f\uff1a<\/p>\n<p>\u2022 \u6807\u51c6\u5316\u6d4b\u8bd5\u6761\u4ef6<br \/>\u2022 \u57fa\u7ebf\u6bd4\u8f83<br \/>\u2022 \u957f\u671f\u968f\u8bbf<br \/>\u2022 \u5ba2\u89c2\u6d4b\u91cf\u5c3a\u5ea6<\/p>\n<p>\u89c6\u9891\u4e2d\u8868\u73b0\u5f97\u66f4\u5f3a\u58ee\u7684\u5b69\u5b50\u53ef\u80fd\u53d7\u5230\u4ee5\u4e0b\u56e0\u7d20\u7684\u5f71\u54cd\uff1a<\/p>\n<p>\u2022 \u6fc0\u52b1\u6216\u9f13\u52b1<br \/>\u2022 \u9009\u62e9\u6027\u5f55\u5236\u6216\u7f16\u8f91<br \/>\u2022 \u81ea\u7136\u7684\u65e5\u5e38\u6ce2\u52a8<\/p>\n<p>\u533b\u751f\u5728\u8fdb\u884c\u4e34\u5e8a\u8bc4\u4f30\u65f6\u4e3b\u8981\u4f9d\u8d56\uff1a<\/p>\n<p>\u2022 \u529f\u80fd\u91cf\u8868\uff08\u4f8b\u5982\uff0cNSAA\uff09<br \/>\u2022 CK\u3001AST\u3001ALT \u7b49\u751f\u7269\u6807\u5fd7\u7269<br \/>\u2022 \u5bf9\u7167\u4e34\u5e8a\u8bd5\u9a8c\u6570\u636e<\/p>\n<p><strong>\u89c6\u9891\u53ef\u4ee5\u8d77\u5230\u6f14\u793a\u4f5c\u7528\uff0c\u4f46\u4e0d\u80fd\u9a8c\u8bc1\u6cbb\u7597\u6548\u679c\u3002.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705412246\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u57fa\u56e0\u6cbb\u7597\u540e\u4e3a\u4f55\u9700\u8981\u62a5\u544aAST\u548cALT\u6c34\u5e73\uff1f<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>AST\uff08\u5929\u51ac\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff09\u548c ALT\uff08\u4e19\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff09\u662f\u53cd\u6620\u809d\u810f\u548c\u808c\u8089\u5065\u5eb7\u7684\u9176\u3002.<\/p>\n<p>\u5b83\u4eec\u5728\u57fa\u56e0\u6cbb\u7597\u540e\u81f3\u5173\u91cd\u8981\uff0c\u56e0\u4e3a\uff1a<\/p>\n<p>\u2022 \u6c34\u5e73\u5347\u9ad8\u53ef\u80fd\u8868\u660e\u809d\u810f\u627f\u53d7\u538b\u529b\u6216\u5b58\u5728\u6bd2\u6027<br \/>\u2022 \u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\uff0c\u5b83\u4eec\u4e5f\u53cd\u6620\u4e86\u808c\u8089\u5206\u89e3\u3002<br \/>\u2022 \u7a81\u7136\u7684\u5cf0\u503c\u53ef\u80fd\u9884\u793a\u7740\u5b89\u5168\u9690\u60a3<\/p>\n<p>\u76d1\u6d4bAST\u548cALT\u6c34\u5e73\u6709\u52a9\u4e8e\uff1a<\/p>\n<p>\u2022 \u8bc4\u4f30\u6cbb\u7597\u5b89\u5168\u6027<br \/>\u2022 \u53ca\u65e9\u53d1\u73b0\u4e0d\u826f\u53cd\u5e94<br \/>\u2022 \u4e86\u89e3\u8eab\u4f53\u9664\u4e86\u53ef\u89c1\u53d8\u5316\u4e4b\u5916\u7684\u5176\u4ed6\u53cd\u5e94<\/p>\n<p><strong>\u5982\u679c\u6ca1\u6709AST\u548cALT\u6570\u636e\uff0c\u5b89\u5168\u6027\u8bc4\u4f30\u662f\u4e0d\u5b8c\u6574\u7684\u3002.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705562117\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u4e3a\u4ec0\u4e48\u5236\u836f\u516c\u53f8\u4e0d\u516c\u5f00\u5206\u4eab\u5b8c\u6574\u7684\u751f\u7269\u6807\u5fd7\u7269\u6570\u636e\uff1f<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u539f\u56e0\u5e76\u975e\u5355\u4e00\uff0c\u4f46\u5e38\u89c1\u56e0\u7d20\u5305\u62ec\uff1a<\/p>\n<p>\u2022 \u76d1\u7ba1\u65f6\u95f4\u8868\uff08\u6570\u636e\u5728\u5ba1\u67e5\u6d41\u7a0b\u540e\u53d1\u5e03\uff09<br \/>\u2022 \u9009\u62e9\u6027\u62a5\u9053\u7b56\u7565\uff08\u5148\u7a81\u51fa\u79ef\u6781\u7ed3\u679c\uff09<br \/>\u2022 \u5bf9\u539f\u59cb\u6570\u636e\u8bef\u8bfb\u7684\u62c5\u5fe7<br \/>\u2022 \u7ade\u4e89\u548c\u5546\u4e1a\u56e0\u7d20<\/p>\n<p>\u7136\u800c\uff0c\u5728\u57fa\u56e0\u7597\u6cd5\u7b49\u9ad8\u98ce\u9669\u6cbb\u7597\u4e2d\uff1a<\/p>\n<p>\u2022 \u900f\u660e\u5ea6\u5e94\u51cc\u9a7e\u4e8e\u8fd9\u4e9b\u62c5\u5fe7\u4e4b\u4e0a\u3002<br \/>\u60a3\u8005\u53ca\u5176\u5bb6\u5c5e\u9700\u8981\u5b8c\u6574\u7684\u4fe1\u606f\uff0c\u800c\u4e0d\u662f\u7247\u9762\u7684\u4fe1\u606f\u3002<\/p>\n<p>\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u7b49\u673a\u6784\u6709\u8d23\u4efb\u786e\u4fdd\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u6570\u636e\uff08\u5305\u62ec\u751f\u7269\u6807\u5fd7\u7269\uff09\u7684\u53ef\u83b7\u53d6\u6027\u548c\u53ef\u9760\u6027\u3002.<\/p>\n\n<\/div>\n<\/div>\n<div id=\"faq-question-1776705628252\" class=\"rank-math-list-item\">\n<h3 class=\"rank-math-question\">\u5728\u8bc4\u4f30\u57fa\u56e0\u7597\u6cd5\u65f6\uff0c\u9664\u4e86\u89c6\u9891\u4e4b\u5916\uff0c\u5bb6\u5ead\u5e94\u8be5\u5173\u6ce8\u54ea\u4e9b\u65b9\u9762\uff1f<\/h3>\n<div class=\"rank-math-answer\">\n\n<p>\u5bb6\u5ead\u5e94\u4f18\u5148\u8003\u8651\u5ba2\u89c2\u7684\u3001\u7ecf\u4e34\u5e8a\u9a8c\u8bc1\u7684\u6570\u636e\uff0c\u5305\u62ec\uff1a<\/p>\n<p>\u2022 \u6cbb\u7597\u524d\u540e CK \u6c34\u5e73\u7684\u53d8\u5316\u8d8b\u52bf<br \/>\u2022 \u7528\u4e8e\u5b89\u5168\u76d1\u6d4b\u7684AST\u548cALT\u503c<br \/>\u2022 \u5bf9\u7167\u4e34\u5e8a\u8bd5\u9a8c\u7684\u7ed3\u679c<br \/>\u2022 \u529f\u80fd\u8bc4\u4f30\uff08\u4f8b\u5982\uff0c, <a href=\"https:\/\/dmdwarrior.com\/zh\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" data-type=\"post\" data-id=\"4959\" rel=\"noreferrer noopener\">NSAA \u5f97\u5206<\/a>)<br \/>\u2022 \u957f\u671f\u968f\u8bbf\u6570\u636e\uff0812-24\u4e2a\u6708\u6216\u66f4\u957f\u65f6\u95f4\uff09<\/p>\n<p>\u6b64\u5916\uff0c\u5bb6\u5ead\u8fd8\u5e94\u8be5\uff1a<\/p>\n<p>\u2022 \u54a8\u8be2\u795e\u7ecf\u808c\u8089\u4e13\u79d1\u533b\u751f<br \/>\u2022 \u67e5\u9605\u6765\u81ea\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u7b49\u53ef\u4fe1\u76d1\u7ba1\u673a\u6784\u7684\u6570\u636e<br \/>\u2022 \u8c28\u614e\u5bf9\u5f85\u4ec5\u57fa\u4e8e\u793e\u4ea4\u5a92\u4f53\u5f97\u51fa\u7684\u7ed3\u8bba<\/p>\n<p><strong>\u5173\u952e\u539f\u5219\uff1a\u51b3\u7b56\u5e94\u57fa\u4e8e\u6570\u636e\uff0c\u800c\u4e0d\u662f\u5b64\u7acb\u7684\u89c6\u89c9\u5370\u8c61\u3002.<\/strong><\/p>\n\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4e3a\u4ec0\u4e48\u5e94\u8be5\u516c\u5f00\u808c\u9178\u6fc0\u9176 (CK)\u3001\u5929\u51ac\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176 (AST)\u3001\u4e19\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176 (ALT) \u548c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"final-thoughts\">\u6700\u540e\u7684\u60f3\u6cd5<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u7597\u6cd5\u7684\u672a\u6765\u53d6\u51b3\u4e8e\u4e00\u4e2a\u539f\u5219\uff1a <strong>\u8bc1\u636e\u5fc5\u987b\u5148\u4e8e\u53d9\u8ff0\u3002<\/strong>.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5f00\u53d1\u7597\u6cd5\u7684\u516c\u53f8\uff08\u5305\u62ec Solid Biosciences (SGT-003)\u3001REGENXBIO (RGX-202)\u3001Genethon (GNT0004) \u548c Satellos (SAT-3247)\uff09\u9884\u8ba1\u5c06\u901a\u8fc7\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\u7b49\u5b98\u65b9\u6e20\u9053\u516c\u5f00\u62ab\u9732\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u57fa\u56e0\u6cbb\u7597\u540e\u7684 CK \u6c34\u5e73\u4ee5\u53ca AST \u548c ALT \u6570\u636e\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u793e\u533a\u4e0d\u9700\u8981\u66f4\u591a\u89c6\u9891\u3002.<br><strong>\u5b83\u9700\u8981\u53ef\u6d4b\u91cf\u3001\u53ef\u9a8c\u8bc1\u548c\u53ef\u4fe1\u8d56\u7684\u6570\u636e\u3002.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u8fbe\u5230\u8fd9\u4e00\u6807\u51c6\u4e4b\u524d\uff0c\u6000\u7591\u4e0d\u662f\u62b5\u6297\uff0c\u800c\u662f\u8d23\u4efb\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"stop-the-noise-just-share-the-data-for-duchenne-muscular-dystrophy\"><strong>\u505c\u6b62\u4f9d\u8d56\u4e0d\u5fc5\u8981\u4e14\u4e0d\u79d1\u5b66\u7684\u89c6\u9891\u2014\u2014\u4e13\u6ce8\u4e8e\u5206\u4eab\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6570\u636e\u3002.<\/strong><\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/dmdwarrior.com\/zh\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4ec5\u975e\u753e\u4f53\u6297\u708e\u836f\uff08NSAA\uff09\u6c34\u5e73\u4e0d\u8db3\u4ee5\u53cd\u6620\u771f\u5b9e\u60c5\u51b5\uff1a\u4e3a\u4f55\u5e94\u62ab\u9732\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u3001\u5929\u51ac\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff08AST\uff09\u3001\u4e19\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\uff08ALT\uff09\u548c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73<\/a><\/p>\n\n\n\n<hr class=\"wp-block-separator has-alpha-channel-opacity\"\/>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"academic-sources-and-references\"><strong>\u5b66\u672f\u8d44\u6e90\u548c\u53c2\u8003\u6587\u732e<\/strong><\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bushby, K.\u3001Finkel, R.\u3001Birnkrant, DJ \u7b49\u3002.<\/strong> \uff082010\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8bca\u65ad\u548c\u6cbb\u7597\uff0c\u7b2c 1 \u90e8\u5206\uff1a\u8bca\u65ad\u3001\u836f\u7269\u6cbb\u7597\u548c\u5fc3\u7406\u793e\u4f1a\u6cbb\u7597\u3002\u300a\u67f3\u53f6\u5200\u795e\u7ecf\u75c5\u5b66\u300b\uff0c9(1)\uff0c77-93\u3002. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(09)70271-6\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(09)70271-6<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Bushby, K.\u3001Finkel, R.\u3001Birnkrant, DJ \u7b49\u3002.<\/strong> \uff082010\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8bca\u65ad\u548c\u6cbb\u7597\uff0c\u7b2c 2 \u90e8\u5206\uff1a\u591a\u5b66\u79d1\u62a4\u7406\u7684\u5b9e\u65bd\u3002\u300a\u67f3\u53f6\u5200\u795e\u7ecf\u75c5\u5b66\u300b\uff0c9(2)\uff0c177\u2013189\u3002. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(09)70272-8\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(09)70272-8<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birnkrant, DJ\u3001Bushby, K.\u3001Bann, CM \u7b49\u3002.<\/strong> \uff082018\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8bca\u65ad\u548c\u6cbb\u7597\uff0c\u7b2c 1 \u90e8\u5206\uff1a\u8bca\u65ad\u3001\u795e\u7ecf\u808c\u8089\u3001\u5eb7\u590d\u3001\u5185\u5206\u6ccc\u548c\u80c3\u80a0\u9053\u6cbb\u7597\u3002\u300a\u67f3\u53f6\u5200\u795e\u7ecf\u75c5\u5b66\u300b\uff0c17(3)\uff0c251\u2013267\u3002. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(18)30024-3\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(18)30024-3<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Birnkrant, DJ\u3001Bushby, K.\u3001Bann, CM \u7b49\u3002.<\/strong> \uff082018\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8bca\u65ad\u548c\u6cbb\u7597\uff0c\u7b2c 2 \u90e8\u5206\uff1a\u547c\u5438\u3001\u5fc3\u810f\u3001\u9aa8\u9abc\u5065\u5eb7\u548c\u9aa8\u79d1\u6cbb\u7597\u3002\u300a\u67f3\u53f6\u5200\u795e\u7ecf\u75c5\u5b66\u300b\uff0c17(4)\uff0c347\u2013361\u3002. <a href=\"https:\/\/doi.org\/10.1016\/S1474-4422(18)30025-5\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/S1474-4422(18)30025-5<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Mendell, JR\u3001Sahenk, Z.\u3001Lehman, K. \u7b49\u4eba\u3002.<\/strong> \uff082020\uff09\u3002\u5bf9\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u60a3\u513f\u8fdb\u884c rAAVrh74.MHCK7.micro-dystrophin \u5168\u8eab\u9012\u9001\u7684\u8bc4\u4f30\u3002\u300a\u65b0\u82f1\u683c\u5170\u533b\u5b66\u6742\u5fd7\u300b\uff0c383(21)\uff0c2039\u20132047\u3002. <a href=\"https:\/\/doi.org\/10.1056\/NEJMoa2002295\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1056\/NEJMoa2002295<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6bb5\uff0cD.<\/strong> \uff082018\uff09\u3002\u7cfb\u7edf\u6027 AAV \u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u57fa\u56e0\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u3002\u5206\u5b50\u6cbb\u7597\uff0c26\uff0810\uff09\uff0c2337\u20132356\u3002. <a href=\"https:\/\/doi.org\/10.1016\/j.ymthe.2018.03.012\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1016\/j.ymthe.2018.03.012<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Hathout, Y., Brody, E., Clemens, PR \u7b49\u3002.<\/strong> \uff082015\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u5927\u89c4\u6a21\u8840\u6e05\u86cb\u767d\u751f\u7269\u6807\u5fd7\u7269\u53d1\u73b0\u3002\u7f8e\u56fd\u56fd\u5bb6\u79d1\u5b66\u9662\u9662\u520a\uff08PNAS\uff09\uff0c112\uff0823\uff09\uff0c7153\u20137158\u3002. <a href=\"https:\/\/doi.org\/10.1073\/pnas.1507719112\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1073\/pnas.1507719112<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>McDonald, CM \u7b49\u3002.<\/strong> \uff082018\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u8840\u6e05\u751f\u7269\u6807\u5fd7\u7269\u3002\u795e\u7ecf\u75c5\u5b66\uff0c90\uff0821\uff09\uff0ce1839\u2013e1848\u3002. <a href=\"https:\/\/doi.org\/10.1212\/WNL.0000000000005515\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1212\/WNL.0000000000005515<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u5e03\u5170\u5361\u4e54\uff0cP.\uff0c\u91cc\u76ae\uff0cG.\uff0c\u548c\u9a6c\u5bcc\u5229\uff0cN.<\/strong> \uff082007\uff09\u3002\u808c\u8089\u635f\u4f24\u7684\u751f\u5316\u6807\u5fd7\u7269\u3002\u4e34\u5e8a\u5316\u5b66\u548c\u5b9e\u9a8c\u5ba4\u533b\u5b66\uff0c45\uff085\uff09\uff0c603-611\u3002. <a href=\"https:\/\/doi.org\/10.1515\/CCLM.2007.115\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1515\/CCLM.2007.115<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nathwani, RA, Pais, S., Reynolds, TB, &amp; Kaplowitz, N.<\/strong> \uff082005\uff09\u3002\u9aa8\u9abc\u808c\u75be\u75c5\u4e2d\u7684\u8840\u6e05\u4e19\u6c28\u9178\u6c28\u57fa\u8f6c\u79fb\u9176\u3002\u809d\u75c5\u5b66\uff0c41\uff082\uff09\uff0c380-382\u3002. <a href=\"https:\/\/doi.org\/10.1002\/hep.20548\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1002\/hep.20548<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>High, KA \u548c Roncarolo, MG.<\/strong> \uff082019\uff09\u3002\u57fa\u56e0\u6cbb\u7597\u3002\u300a\u65b0\u82f1\u683c\u5170\u533b\u5b66\u6742\u5fd7\u300b\uff0c381\uff085\uff09\uff0c455-464\u3002. <a href=\"https:\/\/doi.org\/10.1056\/NEJMra1706910\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1056\/NEJMra1706910<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u95e8\u5fb7\u5c14 (JR) \u548c\u7f57\u8fea\u8bfa-\u514b\u62c9\u5e15\u514b (LR).<\/strong> \uff082020\uff09\u3002\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\uff1aCRISPR\/Cas9 \u6cbb\u7597\u3002\u7ec6\u80de\u7814\u7a76\uff0c30\uff086\uff09\uff0c465\u2013466\u3002. <a href=\"https:\/\/doi.org\/10.1038\/s41422-020-0317-7\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1038\/s41422-020-0317-7<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\u3002.<\/strong> \u57fa\u56e0\u6cbb\u7597\u76d1\u7ba1\u6307\u5357\u548c\u8bc4\u4f30\u62a5\u544a\u3002\u53ef\u8bbf\u95ee\u4ee5\u4e0b\u7f51\u5740\u83b7\u53d6\uff1a <a href=\"https:\/\/www.ema.europa.eu\" target=\"_blank\" rel=\"noopener\">https:\/\/www.ema.europa.eu<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u3002.<\/strong> \u9762\u5411\u884c\u4e1a\u7684\u57fa\u56e0\u6cbb\u7597\u4eba\u7c7b\u6cbb\u7597\u6307\u5357\u3002\u53ef\u8bbf\u95ee\u4ee5\u4e0b\u7f51\u5740\u83b7\u53d6\uff1a <a href=\"https:\/\/www.fda.gov\" target=\"_blank\" rel=\"noopener\">https:\/\/www.fda.gov<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><\/p>","protected":false},"excerpt":{"rendered":"<p>In the evolving landscape of gene therapy for Duchenne muscular dystrophy (DMD), one issue is becoming increasingly clear: CK levels after Duchenne gene therapy are far more important than viral videos circulating online. Families are being exposed to emotionally powerful, time-lapse videos that appear to show improvement after treatment. However, these visual narratives are often [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":7673,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[697,698,696,701,387,522,75,700,68,699,523,61],"class_list":["post-7667","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-alanine-aminotransferase","tag-alt","tag-aspartate-aminotransferase","tag-ast-alt-biomarkers","tag-astrazeneca","tag-biomarkers","tag-ck","tag-ck-levels-duchenne","tag-creatine-kinase","tag-dmd-biomarkers","tag-duchenne-biomarkers","tag-gene-therapy"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/7667","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=7667"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/7667\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/7673"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=7667"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=7667"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=7667"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}