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神经发育障碍的临床试验:治疗前沿。

Clinical trials for neurodevelopmental disorders: At a therapeutic frontier.

机构信息

Center for Autism Research and Treatment, Semel Institute, David Geffen School of Medicine, University of California at Los Angeles (UCLA), Los Angeles, CA 90095-17610, USA.

Center for Autism Research and Treatment, Semel Institute, and Neurogenetics Program, Department of Neurology, David Geffen School of Medicine, UCLA, Los Angeles, CA 90095-1761, USA.

出版信息

Sci Transl Med. 2016 Jan 13;8(321):321fs1. doi: 10.1126/scitranslmed.aad9874.

Abstract

A well-powered clinical trial that failed to replicate promising results in animal models of fragile X syndrome yields important lessons for clinical trial design (Berry-Kravis et al., this issue).

摘要

一项有力的临床试验未能复制脆性 X 综合征动物模型中有希望的结果,为临床试验设计提供了重要的经验教训(Berry-Kravis 等人,本期)。

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