美國食品藥品管理局（FDA）于2018年2月公布了“杜氏肌營養(yǎng)不良及相關(guān)抗肌萎縮蛋白病治療藥物研發(fā)的指導(dǎo)原則”，提出了對(duì)用于支持治療一種或多種抗肌萎縮蛋白病適應(yīng)癥的藥物的臨床研發(fā)方案及臨床試驗(yàn)設(shè)計(jì)的看法。我國目前尚無相關(guān)的指導(dǎo)原則，介紹FDA該指導(dǎo)原則的主要內(nèi)容，以供業(yè)界參考。

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.