Protic, Dragana (18635502600)Dragana (18635502600)ProticAydin, Elber Y. (57209848720)Elber Y. (57209848720)AydinTassone, Flora (7006128050)Flora (7006128050)TassoneTan, Maria M. (57209849735)Maria M. (57209849735)TanHagerman, Randi J. (7006679292)Randi J. (7006679292)HagermanSchneider, Andrea (56910354900)Andrea (56910354900)Schneider2025-06-122025-06-122019https://doi.org/10.1002/mgg3.745https://www.scopus.com/inward/record.uri?eid=2-s2.0-85068917031&doi=10.1002%2fmgg3.745&partnerID=40&md5=c335ba20c89a0f25483b070193071226https://remedy.med.bg.ac.rs/handle/123456789/5549Background: 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. © 2019 The Authors. Molecular Genetics & Genomic Medicine published by Wiley Periodicals, Inc.cognition improvementfragile X syndromemetformintargeted treatment