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J Clin Psychiatry. 2014 Apr;75(4):e294-307. doi: 10.4088/JCP.13r08714.

Translating molecular advances in fragile X syndrome into therapy: a review.

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  • 1Department of Pediatrics and MIND Institute, School of Medicine, University of California, Davis.

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.

© Copyright 2014 Physicians Postgraduate Press, Inc.

PMID:
24813413
[PubMed - indexed for MEDLINE]
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