Fragile X Syndrome: An Update on Developing Treatment Modalities

May 19, 2011

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This review article draws upon more than 100 studies to propose a paradigm shift in the approach to drug development for intellectual disabilities based on a new-found understanding of the core pathophysiology of fragile X syndrome. This paper highlights a number of recent scientific advances in understanding the genetic and biologic causes of fragile X syndrome, which are informing the development of novel therapeutic candidates to address the root cognitive deficits of fragile X. This work may have application to the treatment of other neurodevelopmental disorders.