医学
杜氏肌营养不良
临床试验
肌营养不良
遗传增强
药品审批
物理疗法
重症监护医学
物理医学与康复
基因
内科学
药理学
药品
遗传学
生物
出处
期刊:JAMA
[American Medical Association]
日期:2024-05-01
被引量:1
标识
DOI:10.1001/jama.2024.5613
摘要
This Viewpoint examines the appropriateness of FDA accelerated approval of novel gene therapies to treat boys with Duchenne muscular dystrophy following clinical trials with surrogate outcomes that did not demonstrate net benefits.
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