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.
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Authors | Dragana Protic, Elber Y Aydin, Flora Tassone, Maria M Tan, Randi J Hagerman, Andrea Schneider |
Journal | Molecular genetics & genomic medicine
(Mol Genet Genomic Med)
Vol. 7
Issue 7
Pg. e00745
(07 2019)
ISSN: 2324-9269 [Electronic] United States |
PMID | 31104364
(Publication Type: Case Reports, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't)
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Copyright | © 2019 The Authors. Molecular Genetics & Genomic Medicine published by Wiley Periodicals, Inc. |
Chemical References |
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Topics |
- Adult
- Cognition
(drug effects)
- Fragile X Syndrome
(drug therapy)
- Humans
- Intelligence Tests
- Male
- Metformin
(pharmacology)
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