Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin-two cases
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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Evidence Hub reads this as a case report, done in people. Its evidence level is 5 of 5 (Very low · case / narrative / animal / in vitro / in silico). Case reports, narrative reviews, and animal, cell or computer-model studies give the weakest evidence for human health.
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