Translating molecular advances in fragile x syndrome into therapy: A review

Randi J Hagerman, Vincent Des-Portes, Fabrizio Gasparini, Sébastien Jacquemont, Baltazar Gomez-Mancilla

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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 utili7ed 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.

Original languageEnglish (US)
JournalJournal of Clinical Psychiatry
Volume75
Issue number4
DOIs
StatePublished - 2014

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ASJC Scopus subject areas

  • Arts and Humanities (miscellaneous)
  • Psychiatry and Mental health

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