Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin-two cases

Dragana Protic, Elber Y. Aydin, Flora Tassone, Maria M. Tan, Randi J Hagerman, Andrea Schneider

Research output: Contribution to journalArticle

4 Scopus citations

Abstract

Background: The majority of individuals with fragile X syndrome (FXS) have intellectual disability, behavioral problems, autism, and language deficits. IQ typically declines with age in boys with the full mutation. The results of preclinical studies demonstrated that metformin, a biguanide used to treat type 2 diabetes, rescues multiple phenotypes of FXS in both Drosophila and mouse models. Preliminary studies of patients with FXS demonstrated improvements in behavior. Methods: Here, we present two cases of individuals who have been treated with metformin clinically for one year. Results: Both patients demonstrated significant cognitive and behavioral improvements. They also improved eating habits and normalization of their weight percentiles. Conclusion: Metformin may be a candidate drug for treatment of several types of symptoms in individuals with FXS.

Original languageEnglish (US)
Article numbere00745
JournalMolecular Genetics and Genomic Medicine
Volume7
Issue number7
DOIs
StatePublished - Jul 1 2019

Keywords

  • cognition improvement
  • fragile X syndrome
  • metformin
  • targeted treatment

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics
  • Genetics(clinical)

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