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.

中文翻译：

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FDA 和杜氏肌营养不良症的基因治疗

本观点探讨了 FDA 加速批准新型基因疗法治疗杜氏肌营养不良症男孩的适当性，该疗法是在未显示净效益的替代结果的临床试验后进行的。