This work may not be copied, distributed, displayed, published, reproduced, transmitted, modified, posted, sold, licensed, or used for commercial purposes. By downloading this file, you are agreeing to the publisher’s Terms & Conditions.

Review Article

Translating Molecular Advances in Fragile X Syndrome Into Therapy: A Review

Randi J. Hagerman, MD; Vincent Des-Portes, MD, PhD; Fabrizio Gasparini, PhD; Sébastien Jacquemont, MD; and Baltazar Gomez-Mancilla, MD, PhD

Published: April 15, 2014

Article Abstract

Fragile X syndrome is an inherited disease with cognitive, behavioral, and neurologic manifestations, resulting from a single genetic mutation. A variety of treatments that target individual symptoms of fragile X syndrome are currently utilized with limited efficacy. Research in animal models has resulted in the development of potential novel pharmacologic treatments that target the underlying molecular defect in fragile X syndrome, rather than the resultant symptoms. This review describes recent advances in our understanding of the molecular basis of fragile X syndrome and summarizes the ongoing clinical research programs.

J Clin Psychiatry 2014;75(4):e294-e307

Submitted: August 5, 2013; accepted January 16, 2014 (doi:10.4088/JCP.13r08714).

Corresponding author: Baltazar Gomez-Mancilla, MD, PhD, Department of Neuroscience, Translational Medicine, Novartis Institutes of Biomedical Research, WSJ.210.25.5, CH-4056 Basel, Switzerland (baltazar.gomezmancilla@novartis.com).

Volume: 75

Quick Links: Psychiatry

Continue Reading…

Subscribe to read the entire article

$40.00

Buy this Article as a PDF

References

Sign-up to stay
up-to-date today!

SUBSCRIBE

Already registered? Sign In

Case Report

Safety and Tolerability of Concomitant Intranasal Esketamine Treatment With Irreversible, Nonselective MAOIs: A Case Series

Three cases suggest that concomitant use of intranasal esketamine with an irreversible, nonselective MAOI is safe in...

Read More...