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Metformin for Treatment of Fragile X Syndrome and Other Neurological Disorders

Authors
  • Gantois, Ilse
  • Popic, Jelena
  • Khoutorsky, Arkady
  • Sonenberg, Nahum
Type
Published Article
Journal
Annual Review of Medicine
Publisher
Annual Reviews
Publication Date
Jan 27, 2019
Volume
70
Pages
167–181
Identifiers
DOI: 10.1146/annurev-med-081117-041238
Source
Annual Reviews
Keywords
License
Yellow

Abstract

Fragile X syndrome (FXS) is the most frequent inherited form of intellectual disability and autism spectrum disorder. Loss of the fragile X mental retardation protein, FMRP, engenders molecular, behavioral, and cognitive deficits in FXS patients. Experiments using different animal models advanced our knowledge of the pathophysiology of FXS and led to the discovery of many targets for drug treatments. In this review, we discuss the potential of metformin, an antidiabetic drug approved by the US Food and Drug Administration, to correct core symptoms of FXS and other neurological disorders in humans. We summarize its mechanisms of action in different animal and cellular models and human diseases.

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