{"id":4942,"date":"2025-11-20T19:30:28","date_gmt":"2025-11-20T16:30:28","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4942"},"modified":"2026-03-17T10:55:13","modified_gmt":"2026-03-17T07:55:13","slug":"creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/zh\/creatine-kinase-ck-dystrophin-level-nsaa-score-in-elevidys-gene-therapy\/","title":{"rendered":"Elevidys\u57fa\u56e0\u6cbb\u7597\u4e2d\u7684\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u3001\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u548cNSAA\u8bc4\u5206"},"content":{"rendered":"<p class=\"wp-block-paragraph\">\u63a5\u53d7 ELEVIDYS\uff08delandistrogene moxeparvovec-rokl\uff09\u6cbb\u7597\u7684 DMD \u513f\u7ae5\u808c\u9178\u6fc0\u9176 (CK) \u6c34\u5e73\u4e0b\u964d\u591a\u5c11\u3001NSAA \u8bc4\u5206\u548c\u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u4e0a\u5347\u591a\u5c11\uff0c\u4ecd\u7136\u662f\u4e00\u4e2a\u4ee4\u4eba\u597d\u5947\u7684\u95ee\u9898\u3002ELEVIDYS \u662f\u7531 Sarepta Therapeutics \u5f00\u53d1\u7684\u7528\u4e8e\u6cbb\u7597\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u4e16\u754c\u4e0a\u6700\u6602\u8d35\u7684\u7597\u6cd5\u4e4b\u4e00\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">ELEVIDYS\uff08delandistrogene moxeparvovec-rokl\uff09\u7531Sarepta Therapeutics\u516c\u53f8\u7814\u53d1\uff0c\u662f\u4e00\u79cd\u4e00\u6b21\u6027AAV\uff08\u817a\u76f8\u5173\u75c5\u6bd2\uff09\u57fa\u56e0\u7597\u6cd5\uff0c\u4e8e2023\u5e746\u6708\u5728\u7f8e\u56fd\u83b7\u6279\u7528\u4e8e\u6cbb\u75974-5\u5c81\u53ef\u72ec\u7acb\u884c\u8d70\u7684\u513f\u7ae5DMD\u60a3\u8005\uff0c\u4e4b\u540e\u9002\u5e94\u75c7\u8303\u56f4\u6269\u5927\u3002 <a href=\"https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene\" target=\"_blank\" rel=\"noreferrer noopener\">investorrelations.sarepta.com<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5b83\u7684\u8bbe\u8ba1\u76ee\u7684\u662f\u5c06\u7f16\u7801\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff08\u7ea6138 kDa\uff0c\u800c\u6b63\u5e38\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7ea6\u4e3a427 kDa\uff09\u7684\u57fa\u56e0\u5bfc\u5165\u808c\u8089\u7ec6\u80de\u3002 <a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/37566211\/\" target=\"_blank\" rel=\"noreferrer noopener\">PubMed<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e2d\uff0c\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u7f3a\u5931\u6216\u4e25\u91cd\u7f3a\u4e4f\u4f1a\u5bfc\u81f4\u808c\u8089\u7ec6\u80de\u635f\u4f24\uff1b\u968f\u7740\u808c\u7ea4\u7ef4\u7684\u9000\u5316\uff0c\u808c\u9178\u6fc0\u9176 (CK) \u7b49\u6807\u5fd7\u7269\u6c34\u5e73\u5347\u9ad8\u3002\u56e0\u6b64\uff0c\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u4e2d\u8003\u8651\u7684\u4e24\u79cd\u66ff\u4ee3\u751f\u7269\u6807\u5fd7\u7269\u662f\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>CK\u6c34\u5e73\uff0c\u4f5c\u4e3a\u808c\u8089\u635f\u4f24\/\u6e17\u6f0f\u5165\u8840\u7684\u6807\u5fd7\u7269 &gt; <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\uff1f<\/a><\/li>\n\n\n\n<li>\u808c\u8089\u7ec4\u7ec7\u4e2d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff08\u6216\u5728\u672c\u4f8b\u4e2d\u4e3a\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff09\u7684\u8868\u8fbe\u6c34\u5e73<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"the-core-questions-about-creatine-kinase-ck-dystrophin-level-and-nsaa-score-in-elevidys-gene-therapy\"><strong>\u5173\u4e8e Elevidys \u57fa\u56e0\u6cbb\u7597\u4e2d\u808c\u9178\u6fc0\u9176 (CK)\u3001\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u548c NSAA \u8bc4\u5206\u7684\u6838\u5fc3\u95ee\u9898\uff1a<\/strong><\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u63a5\u53d7 ELEVIDYS \u6cbb\u7597\u7684 DMD \u513f\u7ae5\u60a3\u8005\uff0cCK \u6c34\u5e73\u4e0b\u964d\u591a\u5c11\uff1f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\/\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4e0a\u5347\u591a\u5c11\uff1f<\/strong><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li><strong>\u6cbb\u7597\u540e\u8fbe\u5230\u7684\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u5360\u6b63\u5e38\u4eba\u7c7b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u6bd4\u4f8b\u662f\u591a\u5c11\uff1f<\/strong><\/li>\n<\/ul>\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-core-questions-about-creatine-kinase-ck-dystrophin-level-and-nsaa-score-in-elevidys-gene-therapy\">\u5173\u4e8e Elevidys \u57fa\u56e0\u6cbb\u7597\u4e2d\u808c\u9178\u6fc0\u9176 (CK)\u3001\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u548c NSAA \u8bc4\u5206\u7684\u6838\u5fc3\u95ee\u9898\uff1a<\/a><\/li><li><a href=\"#how-much-micro-dystrophin-is-produced-by-elevidys\">Elevidys \u4ea7\u751f\u591a\u5c11\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff1f<\/a><\/li><li><a href=\"#elevidys-alan-cocuklarda-north-star-ambulatuvar-degerlendirmesi-bazen-nsaa-veya-north-star-olarak-da-adlandirilir-puani-ne-kadar-artti\">\u63a5\u53d7 Elevidys \u6cbb\u7597\u7684\u513f\u7ae5\u7684 NSAA \u8bc4\u5206\u63d0\u9ad8\u4e86\u591a\u5c11\uff1f<\/a><ul><li><a href=\"#what-is-north-star-ambulatory-assessment-nsaa\">\u4ec0\u4e48\u662f\u5317\u6781\u661f\u52a8\u6001\u8bc4\u4f30\uff08NSAA\uff09\uff1f<\/a><\/li><\/ul><\/li><li><a href=\"#what-level-of-improvement-was-observed-in-children-who-were-administered-elevidys\">\u63a5\u53d7 Elevidys \u6cbb\u7597\u7684\u513f\u7ae5\u7684\u75c5\u60c5\u6539\u5584\u7a0b\u5ea6\u5982\u4f55\uff1f<\/a><ul><li><a href=\"#what-we-do-know-about-micro-dystrophin-increase\">\u6211\u4eec\u76ee\u524d\u5bf9\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u589e\u52a0\u7684\u4e86\u89e3<\/a><\/li><li><a href=\"#what-we-do-not-yet-know-clearly-about-ck-decrease\">\u5173\u4e8eCK\u964d\u4f4e\uff0c\u6211\u4eec\u76ee\u524d\u5c1a\u4e0d\u6e05\u695a\u7684\u662f\u54ea\u4e9b\u65b9\u9762\u3002<\/a><\/li><li><a href=\"#why-the-data-gap-matters\">\u6570\u636e\u7f3a\u53e3\u4e3a\u4f55\u5982\u6b64\u91cd\u8981<\/a><\/li><li><a href=\"#what-this-means-for-children-families-and-clinicians\">\u8fd9\u5bf9\u513f\u7ae5\/\u5bb6\u5ead\u548c\u4e34\u5e8a\u533b\u751f\u610f\u5473\u7740\u4ec0\u4e48<\/a><\/li><\/ul><\/li><li><a href=\"#n\">\u5173\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6eb6\u9176\u4f53\u635f\u4f24\u7684\u65b0\u7814\u7a76<\/a><\/li><li><a href=\"#real-world-experience-with-elevidys-gene-therapy-report-from-qatar\">\u6765\u81ea\u5361\u5854\u5c14\u7684Elevidys\u57fa\u56e0\u7597\u6cd5\u5b9e\u9645\u5e94\u7528\u7ecf\u9a8c\u62a5\u544a<\/a><ul><li><a href=\"#alt-and-ast-levels\">ALT \u548c AST \u6c34\u5e73<\/a><\/li><li><a href=\"#creatine-kinase-ck-levels\">\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73<\/a><\/li><li><a href=\"#n-1\">NSAA \u5f97\u5206<\/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=\"how-much-micro-dystrophin-is-produced-by-elevidys\">Elevidys \u4ea7\u751f\u591a\u5c11\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff1f<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u6839\u636eElevidys\u5b98\u65b9\u7f51\u7ad9\u516c\u5e03\u7684\u4fe1\u606f\uff0c\u5728\u6240\u6709\u4e34\u5e8a\u8bd5\u9a8c\u4e2d\uff0c\u53d7\u8bd5\u8005\u5728\u63a5\u53d73\u4e2a\u6708\u6cbb\u7597\u540e\u5e73\u5747\u4ea7\u751f\u4e8634%\u81f351%\u7684ELEVIDYS\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002\u4f46\u8be5\u7f51\u7ad9\u5e76\u672a\u5177\u4f53\u8bf4\u660e\u8fd9\u4e00\u6570\u91cf\u76f8\u5f53\u4e8e\u591a\u5c11\u6b63\u5e38\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><a href=\"https:\/\/dmdwarrior.com\/zh\/fda-rejection-letter-vyondys-53-golodirsen\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u770b\u770bVyondys 53\u5916\u663e\u5b50\u8df3\u8dc3\u7597\u6cd5\u4e2d\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u589e\u52a0\u60c5\u51b5\u3002.<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"elevidys-alan-cocuklarda-north-star-ambulatuvar-degerlendirmesi-bazen-nsaa-veya-north-star-olarak-da-adlandirilir-puani-ne-kadar-artti\">\u63a5\u53d7 Elevidys \u6cbb\u7597\u7684\u513f\u7ae5\u7684 NSAA \u8bc4\u5206\u63d0\u9ad8\u4e86\u591a\u5c11\uff1f<\/h2>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"what-is-north-star-ambulatory-assessment-nsaa\">\u4ec0\u4e48\u662f\u5317\u6781\u661f\u52a8\u6001\u8bc4\u4f30\uff08NSAA\uff09\uff1f<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u603b\u52060-34\u5206\uff0c\u5206\u6570\u8d8a\u9ad8\u8868\u793a\u529f\u80fd\u6c34\u5e73\u8d8a\u9ad8\u3002\u8be5\u8bc4\u4f30\u5305\u62ec\u4e24\u9879\u8ba1\u65f6\u6d4b\u8bd5\uff1a\u5b9a\u65f6\u8d77\u7acb\u6d4b\u8bd5\uff08RFF\uff09\u548c10\u7c73\u5b9a\u65f6\u6b65\u884c\/\u8dd1\u6b65\u6d4b\u8bd5\uff0810MWR\uff09\u3002\u8be5\u8bc4\u4f30\u65b9\u6cd5\u5df2\u901a\u8fc7\u9a8c\u8bc1\uff0c\u5e76\u5e7f\u6cdb\u5e94\u7528\u4e8e\u4e34\u5e8a\u8bd5\u9a8c\u548c\u4e34\u5e8a\u5b9e\u8df5\u4e2d\u3002. <strong>\u9605\u8bfb\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/north-star-ambulatory-assessment-nsaa-in-duchenne-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">NSAA<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6839\u636e Elevidys \u5b98\u65b9\u7f51\u7ad9\u516c\u5e03\u7684\u4fe1\u606f\uff0c\u63a5\u53d7\u57fa\u56e0\u6cbb\u7597\u7684\u513f\u7ae5\u5728 34 \u5206\u5236 NSAA \u6d4b\u8bd5\u4e2d\u5f97\u5206\u63d0\u9ad8\u4e86 2.6 \u5206\uff0c\u800c\u672a\u63a5\u53d7\u57fa\u56e0\u6cbb\u7597\u7684\u513f\u7ae5\u5f97\u5206\u63d0\u9ad8\u4e86 1.9 \u5206\u3002. <strong>\u8fd9\u610f\u5473\u7740\u672a\u63a5\u53d7 Elevidys \u57fa\u56e0\u6cbb\u7597\u7684\u513f\u7ae5\u4e5f\u51fa\u73b0\u4e86\u75c5\u60c5\u6539\u5584\u3002.<\/strong> &gt;&gt;&gt; <a href=\"https:\/\/www.elevidys.com\/about-elevidys\/results-ambulatory\" target=\"_blank\" rel=\"noreferrer noopener\">Elevidys<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6839\u636eEMA\u62a5\u544a\uff0c\u6b64\u6b21\u6da8\u5e45\u4ec5\u4e3a0.65\u4e2a\u767e\u5206\u70b9\u3002 <a href=\"https:\/\/dmdwarrior.com\/zh\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD\u6218\u58eb<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-level-of-improvement-was-observed-in-children-who-were-administered-elevidys\">\u63a5\u53d7 Elevidys \u6cbb\u7597\u7684\u513f\u7ae5\u7684\u75c5\u60c5\u6539\u5584\u7a0b\u5ea6\u5982\u4f55\uff1f<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u63a5\u53d7 ELEVIDYS \u6cbb\u7597\u7684\u60a3\u8005\u5728\u4e00\u5e74\u540e\uff08\u5e73\u5747\u800c\u8a00\uff09\u4ece\u5730\u4e0a\u7ad9\u8d77\u6765\u7684\u901f\u5ea6\u6bd4\u63a5\u53d7\u5b89\u6170\u5242\uff08\u672a\u670d\u7528 ELEVIDYS\uff09\u7684\u60a3\u8005\u5feb 0.64 \u79d2\u3002 <a href=\"https:\/\/www.institut-myologie.org\/en\/2023\/11\/20\/mixed-efficacy-results-for-srp-9001-elevidys-a-gene-therapy-approved-in-the-usa-for-dmd\/\" target=\"_blank\" rel=\"noreferrer noopener\">SRP-9001 (Elevidys) \u7684\u7597\u6548\u7ed3\u679c<\/a><\/li>\n<\/ul>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u63a5\u53d7 ELEVIDYS \u6cbb\u7597\u7684\u60a3\u8005\u5728\u4e00\u5e74\u540e\uff08\u5e73\u5747\u800c\u8a00\uff09\u6b65\u884c\/\u8dd1\u6b65 10 \u7c73\u7684\u901f\u5ea6\u6bd4\u63a5\u53d7\u5b89\u6170\u5242\uff08\u672a\u670d\u7528 ELEVIDYS\uff09\u7684\u60a3\u8005\u5feb 0.42 \u79d2 &gt;&gt;&gt; <a href=\"https:\/\/www.elevidys.com\/about-elevidys\/results-ambulatory\" target=\"_blank\" rel=\"noreferrer noopener\">\u95e8\u8bca\u7ed3\u679c<\/a><\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"what-we-do-know-about-micro-dystrophin-increase\">\u6211\u4eec\u76ee\u524d\u5bf9\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u589e\u52a0\u7684\u4e86\u89e3<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u6839\u636e\u6279\u51c6\u6458\u8981\uff0cFDA \u7814\u7a76\u53d1\u73b0\uff0cELEVIDYS \u53ef\u589e\u52a0\u63a5\u53d7\u6cbb\u7597\u60a3\u8005\u9aa8\u9abc\u808c\u4e2d\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u3002 <a href=\"https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene\" target=\"_blank\" rel=\"noreferrer noopener\">investorrelations.sarepta.com<\/a><\/li>\n\n\n\n<li>\u4e00\u7bc7\u7efc\u8ff0\u6587\u7ae0\uff08\u9884\u5370\u672c\uff09\u6307\u51fa\uff1a\u201c\u7ecf\u8fc7 48 \u5468\u7684\u6cbb\u7597\uff0c\u60a3\u8005\u5728\u86cb\u767d\u8d28\u5370\u8ff9\u5206\u6790\u4e2d\u663e\u793a\u51fa\u5e73\u5747\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u91cf\u7ea6\u4e3a 28 %\u3002\u201d &gt;&gt;&gt; <a href=\"https:\/\/www.preprints.org\/manuscript\/202506.2161\/download\/final_file\" target=\"_blank\" rel=\"noreferrer noopener\">preprints.org<\/a><\/li>\n\n\n\n<li>\u5173\u4e8eEMBARK\u7814\u7a76\u7684\u516c\u544a\u6307\u51fa\uff1a\u2018\u5728\u7b2c\u4e00\u90e8\u5206\u63a5\u53d7\u6cbb\u7597\u7684\u60a3\u8005\u4e2d\uff0c\u7ed9\u836f\u540e64\u5468\u91c7\u96c6\u7684\u6d3b\u68c0\u6837\u672c\u663e\u793a\uff0c\u4e0e\u7b2c12\u5468\u7684\u6d3b\u68c0\u6837\u672c\u76f8\u6bd4\uff0cELEVIDYS\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u6301\u7eed\u7a33\u5b9a\uff0c\u8fd9\u901a\u8fc7\u86cb\u767d\u8d28\u5370\u8ff9\u6cd5\u68c0\u6d4b\u5f97\u51fa\uff0c\u5e76\u4e3a\u89c2\u5bdf\u5230\u7684\u529f\u80fd\u6027\u7ed3\u679c\u63d0\u4f9b\u4e86\u751f\u7269\u5b66\u652f\u6301\u3002\u2019\u7136\u800c\uff0c\u8be5\u516c\u544a\u5e76\u672a\u62ab\u9732\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u4ea7\u91cf\u53ca\u5176\u968f\u65f6\u95f4\u63a8\u79fb\u7684\u7ef4\u6301\u60c5\u51b5\u3002 <a href=\"https:\/\/sarepta.gcs-web.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-results-part-2-embark-study\" target=\"_blank\" rel=\"noreferrer noopener\">sarepta.gcs-web.com<\/a><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u56e0\u6b64\uff0c\u4e00\u9879\u53ef\u7528\u7684\u6570\u636e\u662f\uff0c\u5728\u6cbb\u7597\u7ec4\uff08\u8be5\u5c0f\u578b\u7814\u7a76\uff09\u4e2d\uff0c\u5927\u7ea6 48 \u5468\u65f6\uff0cTP146T \u8868\u8fbe\u91cf\u76f8\u5bf9\u4e8e\u6b63\u5e38\u6c34\u5e73\uff08\u901a\u8fc7\u86cb\u767d\u8d28\u5370\u8ff9\u6cd5\u68c0\u6d4b\uff09\u7ea6\u4e3a 28%\u3002\u7136\u800c\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5728\u8be5\u6d4b\u91cf\u4e2d\uff0c\u201c100%\u201d\u7684\u542b\u4e49\uff08\u6b63\u5e38\u5065\u5eb7\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\uff09\u5e76\u4e0d\u603b\u662f\u5f88\u6e05\u695a\u3002.<\/li>\n\n\n\n<li>\u5728\u66f4\u5927\u89c4\u6a21\u7684\u8bd5\u9a8c\u4e2d\uff0c\u529f\u80fd\u6539\u5584\uff08\u8fd0\u52a8\u7ec8\u70b9\uff09\u5e76\u672a\u8fbe\u5230\u4e3b\u8981\u7ec8\u70b9\u3002 <a href=\"https:\/\/www.medscape.com\/viewarticle\/ema-says-no-duchenne-gene-therapyelevidys-2025a1000jqs\" target=\"_blank\" rel=\"noreferrer noopener\">Medscape<\/a><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u56e0\u6b64\uff0c\u867d\u7136\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u6c34\u5e73\u786e\u5b9e\u4f1a\u589e\u52a0\uff0c\u4f46\u5177\u4f53\u5e45\u5ea6\u5404\u4e0d\u76f8\u540c\uff0c\u957f\u671f\u6301\u4e45\u6027\u4ecd\u5728\u7814\u7a76\u4e2d\uff0c\u8f6c\u5316\u4e3a\u4e34\u5e8a\u76ca\u5904\u4ecd\u4e0d\u786e\u5b9a\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"what-we-do-not-yet-know-clearly-about-ck-decrease\">\u5173\u4e8eCK\u964d\u4f4e\uff0c\u6211\u4eec\u76ee\u524d\u5c1a\u4e0d\u6e05\u695a\u7684\u662f\u54ea\u4e9b\u65b9\u9762\u3002<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u6211\u4eec\u67e5\u9605\u7684\u516c\u5f00\u76d1\u7ba1\u6587\u4ef6\u548c\u51fa\u7248\u7269\u4e2d\uff0c\u6ca1\u6709\u660e\u786e\u516c\u5e03\u7684\u3001\u53ef\u9760\u7684\u6570\u636e\u8868\u660e\uff0c\u60a3\u6709\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u7684\u513f\u7ae5\u5728\u63a5\u53d7 ELEVIDYS \u6cbb\u7597\u540e\uff0c\u808c\u9178\u6fc0\u9176 (CK) \u6c34\u5e73\u4f1a\u4e0b\u964d\u591a\u5c11\u3002\u8981\u70b9\uff1a<\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li>FDA \u6269\u5c55\u6279\u51c6\u58f0\u660e\u4e2d\u63d0\u5230\uff0c\u5728\u8003\u8651\u7684\u6240\u6709\u8bc1\u636e\u4e2d\uff0c\u5305\u62ec\u201c\u808c\u9178\u6fc0\u9176\u6c34\u5e73\u7684\u6539\u5584\u201d\u7b49\u7ec8\u70b9\u6307\u6807\u3002 <a href=\"https:\/\/www.fda.gov\/news-events\/press-announcements\/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy\" target=\"_blank\" rel=\"noreferrer noopener\">\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40<\/a><\/li>\n\n\n\n<li>\u7136\u800c\uff0c\u6211\u4eec\u5728\u540c\u884c\u8bc4\u5ba1\u7684\u6587\u732e\u4e2d\uff08\u81f3\u5c11\u5728\u53ef\u8bbf\u95ee\u7684\u6765\u6e90\u4e2d\uff09\u6ca1\u6709\u627e\u5230\u8bf8\u5982\u201cCK \u5e73\u5747\u5728 Y \u5468\/\u6708\u5185\u4e0b\u964d X%\u201d\u4e4b\u7c7b\u7684\u5df2\u53d1\u8868\u6570\u636e\u3002.<\/li>\n\n\n\n<li>\u8bb8\u591a\u7ed3\u679c\u4ecd\u5c5e\u4e8e\u63a2\u7d22\u6027\u6216\u6b21\u8981\u7ec8\u70b9\uff1bEMBARK \u7814\u7a76\u7684\u4e3b\u8981\u529f\u80fd\u7ec8\u70b9\uff08NSAA\uff09\u5728 52 \u5468\u65f6\u672a\u8fbe\u5230\u7edf\u8ba1\u5b66\u610f\u4e49\uff08\u5dee\u5f02\u7ea6\u4e3a 0.65 \u5206\uff09\u3002 <a href=\"https:\/\/www.medscape.com\/viewarticle\/ema-says-no-duchenne-gene-therapyelevidys-2025a1000jqs\" target=\"_blank\" rel=\"noreferrer noopener\">Medscape<\/a><\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\">\u56e0\u6b64\uff0c\u867d\u7136\u6709\u8ff9\u8c61\u8868\u660e CK \u6c34\u5e73\u53ef\u80fd\u4f1a\u4e0b\u964d\uff0c\u4f46\u4e0b\u964d\u7684\u7a0b\u5ea6\uff08\u4f8b\u5982\uff0c\u767e\u5206\u6bd4\u51cf\u5c11\u3001\u7edd\u5bf9\u5355\u4f4d\uff09\u5728\u591a\u9879\u5927\u578b\u7814\u7a76\u4e2d\u5c1a\u672a\u5f97\u5230\u516c\u5f00\u7684\u5145\u5206\u63cf\u8ff0\uff08\u6216\u8005\u81f3\u5c11\u8fd8\u6ca1\u6709\u4ee5\u6613\u4e8e\u83b7\u53d6\u7684\u5f62\u5f0f\u53d1\u8868\uff09\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"why-the-data-gap-matters\">\u6570\u636e\u7f3a\u53e3\u4e3a\u4f55\u5982\u6b64\u91cd\u8981<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u808c\u9178\u6fc0\u9176 (CK) \u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u4e2d\u5e7f\u6cdb\u4f7f\u7528\u7684\u751f\u7269\u6807\u5fd7\u7269\uff0c\u7528\u4e8e\u8ffd\u8e2a\u808c\u8089\u635f\u4f24\/\u6e17\u6f0f\u60c5\u51b5\u3002CK \u6c34\u5e73\u663e\u8457\u4e0b\u964d\u53ef\u80fd\u63d0\u793a\u6301\u7eed\u6027\u808c\u8089\u635f\u4f24\u6709\u6240\u51cf\u8f7b\u3002.<\/li>\n\n\n\n<li>\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff08\u6216\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff09\u7684\u8868\u8fbe\u662f\u4e00\u79cd\u673a\u5236\u6807\u5fd7\uff1a\u8868\u660e\u8be5\u7597\u6cd5\u6b63\u5728\u53d1\u6325\u5176\u8bbe\u8ba1\u7684\u4f5c\u7528\u3002.<\/li>\n\n\n\n<li>\u4f46\u5373\u4f7f\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u589e\u52a0\u800c CK \u51cf\u5c11\uff0c\u5173\u952e\u95ee\u9898\u4ecd\u7136\u5b58\u5728\uff1a <strong>\u8fd9\u662f\u5426\u80fd\u8f6c\u5316\u4e3a\u6709\u610f\u4e49\u7684\u529f\u80fd\/\u8fd0\u52a8\u65b9\u9762\u7684\u76ca\u5904\u3001\u66f4\u957f\u7684\u884c\u8d70\u65f6\u95f4\u3001\u66f4\u9ad8\u7684\u751f\u6d3b\u8d28\u91cf\uff1f<\/strong><\/li>\n\n\n\n<li>\u5927\u578b\u4e09\u671f\u4e34\u5e8a\u8bd5\u9a8c\u672a\u80fd\u8fbe\u5230\u5176\u4e3b\u8981\u8fd0\u52a8\u7ec8\u70b9\uff0c\u8fd9\u4e00\u4e8b\u5b9e\u4ee4\u4eba\u62c5\u5fe7\uff1a\u86cb\u767d\u8d28\u8868\u8fbe\u589e\u52a0\u5e76\u4e0d\u4e00\u5b9a\u80fd\u4fdd\u8bc1\u529f\u80fd\u663e\u8457\u6539\u5584\u3002 <a href=\"https:\/\/www.medscape.com\/viewarticle\/ema-says-no-duchenne-gene-therapyelevidys-2025a1000jqs\" target=\"_blank\" rel=\"noreferrer noopener\">Medscape<\/a><\/li>\n\n\n\n<li>\u76d1\u7ba1\u673a\u6784\uff0c\u4f8b\u5982\u6b27\u6d32\u836f\u54c1\u7ba1\u7406\u5c40\uff08EMA\uff09\uff0c\u5219\u6301\u66f4\u4e3a\u6000\u7591\u7684\u6001\u5ea6\uff1aEMA \u7684\u4eba\u7528\u836f\u54c1\u59d4\u5458\u4f1a\uff08CHMP\uff09\u5efa\u8bae\u5728 2025 \u5e74 7 \u6708\u4e0d\u6388\u4e88 Elevidys \u4e0a\u5e02\u8bb8\u53ef\uff0c\u7406\u7531\u662f\u5c3d\u7ba1\u8be5\u836f\u7269\u80fd\u4ea7\u751f\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\uff0c\u4f46\u5176\u529f\u80fd\u83b7\u76ca\u4e0d\u8db3\u3002 <a href=\"https:\/\/dmdwarrior.com\/zh\/why-elevidys-was-not-approved-by-the-european-medicines-agency-ema\/\" target=\"_blank\" rel=\"noreferrer noopener\">DMD\u6218\u58eb<\/a><\/li>\n<\/ul>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"what-this-means-for-children-families-and-clinicians\">\u8fd9\u5bf9\u513f\u7ae5\/\u5bb6\u5ead\u548c\u4e34\u5e8a\u533b\u751f\u610f\u5473\u7740\u4ec0\u4e48<\/h3>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u5982\u679c\u60a8\u662f\u6b63\u5728\u8003\u8651\u4f7f\u7528 ELEVIDYS \u7684\u5bb6\u5ead\u6216\u4e34\u5e8a\u533b\u751f\uff0c\u90a3\u4e48\u60a8\u5e94\u8be5\u4e86\u89e3\uff0c\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u7684\u8868\u8fbe\u786e\u5b9e\u4f1a\u589e\u52a0\uff08\u4f8b\u5982\uff0c\u5728\u4e00\u9879\u7814\u7a76\u4e2d\uff0c~28%\uff09\uff0c\u4f46\u957f\u671f\u5b9e\u9645\u4f7f\u7528\u4e2d\u80fd\u5426\u8f6c\u5316\u4e3a\u6e05\u6670\u3001\u6709\u610f\u4e49\u7684\u529f\u80fd\u6027\u83b7\u76ca\u4ecd\u6709\u5f85\u9a8c\u8bc1\u3002.<\/li>\n\n\n\n<li>\u7531\u4e8e CK \u964d\u4f4e\u7684\u6570\u503c\u6ca1\u6709\u660e\u786e\u5b9a\u4e49\/\u6ca1\u6709\u4e00\u81f4\u516c\u5e03\uff0c\u56e0\u6b64\u4e0d\u5e94\u5047\u8bbe CK \u4f1a\u4e0b\u964d\u7279\u5b9a\u7684\u767e\u5206\u6bd4\uff1b\u76d1\u6d4b CK \u4ecd\u7136\u662f\u7814\u7a76\/\u4e34\u5e8a\u968f\u8bbf\u7684\u4e00\u90e8\u5206\uff0c\u4f46\u5e94\u8be5\u964d\u4f4e\u9884\u671f\u3002.<\/li>\n\n\n\n<li>\u53c2\u4e0e\u6216\u8fdb\u884c\u540e\u7eed\/\u786e\u8ba4\u6027\u8bd5\u9a8c\uff08\u5982 EMBARK\uff09\u4ee5\u53ca\u76d1\u6d4b\u808c\u8089\u751f\u7269\u6807\u5fd7\u7269\uff08\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u3001CK\uff09\u548c\u529f\u80fd\u7ec8\u70b9\uff08\u884c\u8d70\u3001\u8ba1\u65f6\u6d4b\u8bd5\u3001\u547c\u5438\/\u5fc3\u810f\u72b6\u6001\uff09\u4ecd\u7136\u81f3\u5173\u91cd\u8981\u3002.<\/li>\n\n\n\n<li>\u7531\u4e8e\u57fa\u56e0\u7597\u6cd5\u662f\u4e00\u6b21\u6027\u7684\uff0c\u800c\u4e14\u975e\u5e38\u6602\u8d35\uff08\u636e\u8ba8\u8bba\u62a5\u9053\uff0c\u521d\u59cb\u5b9a\u4ef7\u5728\u6570\u767e\u4e07\u7f8e\u5143\u8303\u56f4\u5185\uff09\uff0c\u5e76\u4e14\u5b58\u5728\u5df2\u77e5\u7684\u5b89\u5168\u98ce\u9669\uff08\u4f8b\u5982\u809d\u635f\u4f24\uff09\uff0c\u56e0\u6b64\u5fc5\u987b\u6743\u8861\u5df2\u77e5\u548c\u672a\u77e5\u56e0\u7d20\u3002.<\/li>\n<\/ul>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"n\">\u5173\u4e8e\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u6eb6\u9176\u4f53\u635f\u4f24\u7684\u65b0\u7814\u7a76<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5c3d\u7ba1\u57fa\u56e0\u7597\u6cd5\uff08delandistrogene moxeparvovec\uff09\u5df2\u83b7\u5f97\u7f8e\u56fd\u98df\u54c1\u836f\u54c1\u76d1\u7763\u7ba1\u7406\u5c40\uff08FDA\uff09\u6279\u51c6\uff08FDA\uff09\u7528\u4e8e\u95e8\u8bca\u60a3\u8005\uff0c\u4f46\u60a3\u8005\u7684\u529f\u80fd\u6539\u5584\u7a0b\u5ea6\u5e76\u672a\u8fbe\u5230\u4e34\u5e8a\u524d\u9884\u671f\u3002\u8fd9\u79cd\u6709\u9650\u7684\u4e34\u5e8a\u83b7\u76ca\u53ef\u80fd\u5f52\u56e0\u4e8e\u591a\u79cd\u56e0\u7d20\uff0c\u5305\u62ec\u7f3a\u4e4f\u5168\u957f\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4e2d\u5b58\u5728\u7684\u67d0\u4e9b\u529f\u80fd\u57df\u3002\u56e0\u6b64\uff0c\u57fa\u56e0\u8f6c\u79fb\u53ef\u80fd\u65e0\u6cd5\u9006\u8f6c\u808c\u8089\u4e2d\u7684\u6240\u6709\u75c5\u7406\u673a\u5236\u3002\u8bc6\u522b\u57fa\u56e0\u7597\u6cd5\u65e0\u6cd5\u7ea0\u6b63\u7684\u7279\u5b9a\u75c5\u7406\u673a\u5236\uff0c\u5e76\u63a2\u7d22\u9488\u5bf9\u8fd9\u4e9b\u673a\u5236\u7684\u8054\u5408\u7597\u6cd5\uff0c\u81f3\u5173\u91cd\u8981\u3002&gt;&gt;&gt; \u9605\u8bfb\u66f4\u591a\uff1a <a href=\"https:\/\/www.science.org\/doi\/10.1126\/sciadv.adv6805\" target=\"_blank\" rel=\"noreferrer noopener\">\u6eb6\u9176\u4f53\u635f\u4f24\u662f\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7\u7684\u6cbb\u7597\u9776\u70b9\u3002<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"real-world-experience-with-elevidys-gene-therapy-report-from-qatar\">\u6765\u81ea\u5361\u5854\u5c14\u7684Elevidys\u57fa\u56e0\u7597\u6cd5\u5b9e\u9645\u5e94\u7528\u7ecf\u9a8c\u62a5\u544a<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u5361\u5854\u5c14\u8fdb\u884c\u7684\u8fd9\u9879\u7814\u7a76\u4e2d\uff0c\u5bf9\u63a5\u53d7\u675c\u6c0f\u808c\u8425\u517b\u4e0d\u826f\u75c7 (DMD) \u57fa\u56e0\u6cbb\u7597\u7684 8 \u540d\u60a3\u8005\u8fdb\u884c\u4e86 30 \u5468\u7684\u968f\u8bbf\uff0c\u76d1\u6d4b\u4e86\u5404\u79cd\u751f\u7269\u6807\u5fd7\u7269\uff08AST\u3001ALT\u3001CK\u3001\u808c\u9499\u86cb\u767d I \u548c GGT\uff09\u7684\u6c34\u5e73\u3002. <strong>\u6765\u6e90<\/strong>: <a href=\"https:\/\/www.nature.com\/articles\/s41434-025-00580-3\" target=\"_blank\" rel=\"noopener\">Nature.com<\/a><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u6211\u4eec\u4e0e\u60a8\u5206\u4eab\u8fd9\u9879\u7814\u7a76\u7684\u4e00\u4e9b\u4eae\u70b9\uff1a<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"alt-and-ast-levels\">ALT \u548c AST \u6c34\u5e73<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">ALT \u548c AST \u6c34\u5e73\u5728\u7b2c\u4e00\u5468\u6709\u6240\u4e0b\u964d\uff0c\u968f\u540e\u51fa\u73b0\u8f7b\u5fae\u6ce2\u52a8\uff0c\u5e76\u5728\u7b2c 2-3 \u5468\u5de6\u53f3\u7565\u6709\u4e0a\u5347\uff0c\u4e4b\u540e\u4ece\u7b2c 4 \u5468\u5f00\u59cb\u8d8b\u4e8e\u7a33\u5b9a\u3002\u4ece\u7b2c 14 \u5468\u5f00\u59cb\uff0c\u5e74\u8f7b\u60a3\u8005\u7684 ALT \u548c AST \u6c34\u5e73\u9010\u6e10\u5347\u9ad8\uff0c\u5728\u7b2c 26 \u5468\u5de6\u53f3\u8fbe\u5230\u5cf0\u503c\uff0c\u4e4b\u540e\u56de\u843d\u81f3\u8f93\u6ce8\u540e\u7b2c\u4e8c\u5468\u5de6\u53f3\u7684\u521d\u59cb\u6c34\u5e73\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"creatine-kinase-ck-levels\">\u808c\u9178\u6fc0\u9176\uff08CK\uff09\u6c34\u5e73<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u5728\u968f\u8bbf\u671f\u95f4\uff0cCK \u7684\u521d\u59cb\u53cd\u5e94\u6700\u4e3a\u663e\u8457\uff0c\u5728\u7b2c\u4e00\u5468\u5c31\u51fa\u73b0\u4e86 44.1% \u7684\u6025\u5267\u4e0b\u964d\u3002\u968f\u540e CK \u6c34\u5e73\u6301\u7eed\u4e0b\u964d\uff0c\u5e76\u5728\u7b2c\u56db\u5468\u5de6\u53f3\u8fbe\u5230\u6700\u4f4e\u70b9\u3002. <strong>\u7136\u800c\uff0c\u5230\u7b2c 7 \u5468\u65f6\uff0cCK \u6c34\u5e73\u663e\u8457\u5347\u9ad8\u4e86 84.2%\u3002.<\/strong> \u8fd9\u79cd\u6a21\u5f0f\u8868\u660e\uff0c\u6cbb\u7597\u521d\u671f\u53cd\u5e94\u5f3a\u70c8\uff0c\u53ef\u80fd\u53cd\u6620\u4e86\u808c\u8089\u4fee\u590d\u6216\u808c\u8089\u635f\u4f24\u51cf\u5c11\uff0c\u968f\u540e\u968f\u7740\u8eab\u4f53\u9002\u5e94\uff0c\u4f1a\u51fa\u73b0\u6ce2\u52a8\u9636\u6bb5\u3002.<\/p>\n\n\n\n<h3 class=\"wp-block-heading\" id=\"n-1\">NSAA \u5f97\u5206<\/h3>\n\n\n\n<p class=\"wp-block-paragraph\">\u6240\u6709\u5bb6\u957f\u5747\u62a5\u544a\u60a3\u8005\u5728\u63a5\u53d7\u57fa\u56e0\u6cbb\u7597\u540e\u4e24\u4e2a\u6708\u5185\u529f\u80fd\u6709\u6240\u6539\u5584\uff0c\u4f46\u4e94\u5206\u4e4b\u56db\u7684\u5bb6\u957f\u8868\u793a\u62c5\u5fc3\u6cbb\u7597\u540e\u4f53\u91cd\u589e\u52a0\u3002\u4e00\u540d\u60a3\u8005\u4ecd\u65e0\u6cd5\u884c\u8d70\uff0c\u65e0\u6cd5\u8fdb\u884c10\u5206\u949f\u6b65\u884c\u6d4b\u8bd5\u6216\u8d77\u7acb\u6d4b\u8bd5\uff1b\u5176\u4ed6\u60a3\u8005\u4e2d\uff0c\u4e00\u540d\u60a3\u8005\u768410\u5206\u949f\u6b65\u884c\u6d4b\u8bd5\u901f\u5ea6\u5728\u4e24\u4e2a\u6708\u5185\u9010\u6e10\u63d0\u9ad8\uff0c\u4e24\u540d\u60a3\u8005\u7684\u901f\u5ea6\u4e0b\u964d\uff0c\u4e00\u540d\u60a3\u8005\u7684\u901f\u5ea6\u4fdd\u6301\u4e0d\u53d8\u3002\u8fd9\u4e9b\u7ed3\u679c\u7ed3\u5408\u5bb6\u957f\u7684\u89c2\u5bdf\uff0c\u63d0\u793a\u7c7b\u56fa\u9187\u5f15\u8d77\u7684\u4f53\u91cd\u589e\u52a0\u53ef\u80fd\u5f71\u54cd\u60a3\u8005\u57fa\u56e0\u6cbb\u7597\u540e\u7684\u529f\u80fd\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">\u9700\u8981\u66f4\u591a\u66f4\u5927\u89c4\u6a21\u7684\u771f\u5b9e\u4e16\u754c\u6570\u636e\u6765\u8fdb\u4e00\u6b65\u8bc1\u5b9e Delandistrogene moxeparvovec \u6cbb\u7597 DMD \u60a3\u8005\u7684\u5b89\u5168\u6027\u548c\u6709\u6548\u6027\u3002.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>: <a href=\"https:\/\/dmdwarrior.com\/zh\/biomarkers-in-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u4ec5\u9760\u975e\u753e\u4f53\u6297\u708e\u836f\uff08NSAA\uff09\u68c0\u6d4b\u662f\u4e0d\u591f\u7684\uff1a\u4e3a\u4ec0\u4e48\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\u5e94\u8be5\u516c\u5f00<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"conclusion\">\u7ed3\u8bba<\/h2>\n\n\n\n<ul class=\"wp-block-list\">\n<li>\u662f\u7684\uff0c\u4f7f\u7528 ELEVIDYS \u6cbb\u7597\u4f1a\u5bfc\u81f4\u808c\u8089\u4e2d\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u8868\u8fbe\u589e\u52a0\uff08\u5728\u4e00\u9879\u7814\u7a76\u4e2d\uff0c\u6570\u503c\u7ea6\u4e3a ~28%\uff09\u3002.<\/li>\n\n\n\n<li>CK \u964d\u4f4e\u7684\u7a0b\u5ea6\u5c1a\u672a\u5f97\u5230\u5145\u5206\u5ba3\u4f20\uff0c\u4e5f\u6ca1\u6709\u5728\u540c\u884c\u8bc4\u5ba1\u7684\u5927\u89c4\u6a21\u7814\u7a76\u4e2d\u5f97\u5230\u5145\u5206\u63cf\u8ff0\u2014\u2014\u56e0\u6b64\uff0c\u5b83\u4ecd\u7136\u662f\u4e00\u4e2a\u201c\u4ee4\u4eba\u597d\u5947\u7684\u95ee\u9898\u201d\u3002.<\/li>\n\n\n\n<li>\u957f\u671f\u529f\u80fd\u83b7\u76ca\u4ecd\u6709\u5f85\u9a8c\u8bc1\uff0c\u8fd9\u610f\u5473\u7740\u751f\u7269\u6807\u5fd7\u7269\u7684\u6539\u5584\uff08\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u5347\u9ad8\uff0cCK \u53ef\u80fd\u964d\u4f4e\uff09\u4ee4\u4eba\u9f13\u821e\uff0c\u4f46\u8fd8\u4e0d\u80fd\u6700\u7ec8\u4fdd\u8bc1\u8fd0\u52a8\u7ed3\u679c\u7684\u6539\u5584\u3002.<\/li>\n\n\n\n<li>\u968f\u7740\u6765\u81ea\u9a8c\u8bc1\u6027\u8bd5\u9a8c\u548c\u771f\u5b9e\u4e16\u754c\u6ce8\u518c\u7814\u7a76\u7684\u66f4\u591a\u6570\u636e\u7684\u51fa\u73b0\uff0c\u6211\u4eec\u9884\u8ba1\u4f1a\u770b\u5230 CK \u964d\u4f4e\u3001\u6301\u7eed\u5b58\u5728\u7684\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\/\u5fae\u578b\u808c\u8425\u517b\u4e0d\u826f\u86cb\u767d\u4ee5\u53ca\u4e0e\u8fd0\u52a8\u529f\u80fd\u7684\u76f8\u5173\u6027\u7684\u66f4\u8be6\u7ec6\u7684\u6570\u636e\u2014\u2014\u8fd9\u5c06\u6709\u52a9\u4e8e\u5bb6\u5ead\u548c\u4e34\u5e8a\u533b\u751f\u505a\u51fa\u66f4\u660e\u667a\u7684\u51b3\u5b9a\u3002.<\/li>\n<\/ul>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>\u4e86\u89e3\u66f4\u591a<\/strong>:&nbsp;<a href=\"https:\/\/dmdwarrior.com\/zh\/frequently-asked-questions-about-elevidys-used-for-duchenne-muscular-dystrophy\/\" target=\"_blank\" rel=\"noreferrer noopener\">\u5173\u4e8e Elevidys \u7684\u5e38\u89c1\u95ee\u9898\u89e3\u7b54<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>It remains a matter of curiosity how much Creatine Kinase (CK) levels decrease, NSAA score and normal dystrophin levels increase in children with DMD who receive ELEVIDYS (delandistrogene moxeparvovec-rokl), one of the world&#8217;s most expensive therapies developed by Sarepta Therapeutics for Duchenne muscular dystrophy. ELEVIDYS (delandistrogene moxeparvovec-rokl), developed by Sarepta Therapeutics, is a one-time AAV [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":5211,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[121,33,179,203,65],"class_list":["post-4942","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-delandistrogene-moxeparvovec","tag-elevidys","tag-elevidys-gene-therapy","tag-roche","tag-sarepta"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4942","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=4942"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/posts\/4942\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media\/5211"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/media?parent=4942"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/categories?post=4942"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/zh\/wp-json\/wp\/v2\/tags?post=4942"}],"curies":[{"name":"\u53ef\u6e7f\u6027\u7c89\u5242","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}