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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.

SUBMITTER: Protic D

PROVIDER: S-EPMC6625129 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature

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

Protic Dragana D Aydin Elber Y EY Tassone Flora F Tan Maria M MM Hagerman Randi J RJ Schneider Andrea A

Molecular genetics & genomic medicine 20190518 7

<h4>Background</h4>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.<h4>Methods</h4>Here ...[more]

PMID: 31104364

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