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[摘要]
美国食品药品管理局(FDA)于2018年2月公布了“杜氏肌营养不良及相关抗肌萎缩蛋白病治疗药物研发的指导原则”,提出了对用于支持治疗一种或多种抗肌萎缩蛋白病适应症的药物的临床研发方案及临床试验设计的看法。我国目前尚无相关的指导原则,介绍FDA该指导原则的主要内容,以供业界参考。
[Key word]
[Abstract]
FDA announced Duchenne muscular dystrophy and related dystrophinopathies developing drugs for treatment guidance for industry in February 2018. This guidance addressed FDA's current thinking regarding clinical development programs and trial designs for drugs to support an indication for the treatment of one or more dystrophinopathies. There is no related guidance in China. We introduce the main content of the guideline so as to provide the reference for corresponding fields.
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