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1	Metformin treatment in young children with fragile X syndrome.
	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
	In: Molecular genetics & genomic medicine, vol 7, iss 11 (2019)
	Abstract: BackgroundMetformin 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.MethodsHere, we present nine children with FXS between 2 and 7years of age who were treated clinically with metformin and monitored for behavioral and metabolic changes.ResultsParent 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.ConclusionThese results support the need for a controlled trial of metformin in children with FXS under 7years old whose brains are in a critical developmental window and thus may experience a greater degree of clinical benefit from metformin.
	Keyword: 6.1 Pharmaceuticals; Basic Behavioral and Social Science; Behavioral and Social Science; Brain Disorders; Child; Clinical Sciences; Diabetes; FMR1; Fragile X Syndrome; Genetics; Humans; Hypoglycemic Agents; Intellectual and Developmental Disabilities (IDD); Male; Medicinal and Biomolecular Chemistry; Metabolic and endocrine; Metformin; Neurodevelopmental Disorders; Orphan Drug; Pediatric; Preschool; Prognosis; Rare Diseases; targeted treatments; translational medicine
	URL: https://escholarship.org/uc/item/6vz0b2jm
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