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Article 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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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6625129 | PMC |
| http://dx.doi.org/10.1002/mgg3.745 | DOI Listing |
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