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Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin-two cases
Dragana Protic1,2, Elber Y Aydin1,3, Flora Tassone1,4
1Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, University of California Davis, Sacramento, California.
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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