Clinical trials for neurodevelopmental disorders: At a therapeutic frontier

Author:

Jeste Shafali S.1,Geschwind Daniel H.2

Affiliation:

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.

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).

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference10 articles.

1. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials

2. R. J. Hagerman The physical and behavioral phenotype in Fragile X Syndrome: Diagnosis Treatment and Research R. J. Hagerman P. Hagerman Eds. (Johns Hopkins Univ. Press. Baltimore 2002) pp. 3–109.

3. Molecular Mechanisms of Fragile X Syndrome: A Twenty-Year Perspective

4. The mGluR theory of fragile X mental retardation

5. The challenges of clinical trials in fragile X syndrome

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