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Metformin treatment in young children with fragile X syndrome.
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In: Molecular genetics & genomic medicine, vol 7, iss 11 (2019)
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A Randomized Controlled Trial of Sertraline in Young Children With Autism Spectrum Disorder.
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A Randomized Controlled Trial of Sertraline in Young Children With Autism Spectrum Disorder.
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A Randomized Controlled Trial of Sertraline in Young Children With Autism Spectrum Disorder
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Metformin treatment in young children with fragile X syndrome
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Biag, Hazel Maridith B.; Potter, Laura A.; Wilkins, Victoria; Afzal, Sumra; Rosvall, Alexis; Salcedo‐Arellano, Maria Jimena; Rajaratnam, Akash; Manzano‐Nunez, Ramiro; Schneider, Andrea; Tassone, Flora; Rivera, Susan M.; Hagerman, Randi J.. - : John Wiley and Sons Inc., 2019
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Abstract:
BACKGROUND: Metformin is a drug commonly used in individuals with type 2 diabetes, obesity, and impaired glucose tolerance. It has a strong safety profile in both children and adults. Studies utilizing the Drosophila model and knock out mouse model of fragile X syndrome (FXS) have found metformin to rescue memory, social novelty deficits, and neuroanatomical abnormalities. These studies provided preliminary evidence that metformin could be used as a targeted treatment for the cognitive and behavioral problems associated with FXS. Previously, a case series of children and adults with FXS treated with metformin demonstrated improvements in irritability, social responsiveness, language, and hyperactivity. METHODS: Here, we present nine children with FXS between 2 and 7 years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes. RESULTS: Parent reports and developmental testing before and after metformin are presented. There were improvements in language development and behavior (such as lethargy and stereotypy) in most of the patients. CONCLUSION: These results support the need for a controlled trial of metformin in children with FXS under 7 years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.
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Original Articles
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URL: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6825840/
https://doi.org/10.1002/mgg3.956
http://www.ncbi.nlm.nih.gov/pubmed/31520524
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