Clinical trials for neurodevelopmental disorders: At a therapeutic frontier

Shafali S Jeste; Daniel H Geschwind

doi:10.1126/scitranslmed.aad9874

Clinical trials for neurodevelopmental disorders: At a therapeutic frontier

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

Authors

Shafali S Jeste¹, Daniel H Geschwind²

Affiliations

¹ 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.
² 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. dhg@mednet.ucla.edu.

PMID: 26764154
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).

Publication types

Review
Comment

MeSH terms

Animals
Fragile X Mental Retardation Protein
Fragile X Syndrome / drug therapy*
Humans
Neurodevelopmental Disorders*

Substances

Fragile X Mental Retardation Protein