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 u...

Full description

Bibliographic Details
Main Authors: Dragana Protic, Elber Y. Aydin, Flora Tassone, Maria M. Tan, Randi J. Hagerman, Andrea Schneider
Format: Article
Language:English
Published: Wiley 2019-07-01
Series:Molecular Genetics & Genomic Medicine
Subjects:
Online Access:https://doi.org/10.1002/mgg3.745
_version_ 1828530181304745984
author Dragana Protic
Elber Y. Aydin
Flora Tassone
Maria M. Tan
Randi J. Hagerman
Andrea Schneider
author_facet Dragana Protic
Elber Y. Aydin
Flora Tassone
Maria M. Tan
Randi J. Hagerman
Andrea Schneider
author_sort Dragana Protic
collection DOAJ
description 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.
first_indexed 2024-12-11T22:19:31Z
format Article
id doaj.art-1ef6e582f9a0486baf251ec14b901cff
institution Directory Open Access Journal
issn 2324-9269
language English
last_indexed 2024-12-11T22:19:31Z
publishDate 2019-07-01
publisher Wiley
record_format Article
series Molecular Genetics & Genomic Medicine
spelling doaj.art-1ef6e582f9a0486baf251ec14b901cff2022-12-22T00:48:29ZengWileyMolecular Genetics & Genomic Medicine2324-92692019-07-0177n/an/a10.1002/mgg3.745Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two casesDragana Protic0Elber Y. Aydin1Flora Tassone2Maria M. Tan3Randi J. Hagerman4Andrea Schneider5Medical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Sacramento CaliforniaAbstract 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.https://doi.org/10.1002/mgg3.745cognition improvementfragile X syndromemetformintargeted treatment
spellingShingle Dragana Protic
Elber Y. Aydin
Flora Tassone
Maria M. Tan
Randi J. Hagerman
Andrea Schneider
Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases
Molecular Genetics & Genomic Medicine
cognition improvement
fragile X syndrome
metformin
targeted treatment
title Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases
title_full Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases
title_fullStr Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases
title_full_unstemmed Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases
title_short Cognitive and behavioral improvement in adults with fragile X syndrome treated with metformin‐two cases
title_sort cognitive and behavioral improvement in adults with fragile x syndrome treated with metformin two cases
topic cognition improvement
fragile X syndrome
metformin
targeted treatment
url https://doi.org/10.1002/mgg3.745
work_keys_str_mv AT draganaprotic cognitiveandbehavioralimprovementinadultswithfragilexsyndrometreatedwithmetformintwocases
AT elberyaydin cognitiveandbehavioralimprovementinadultswithfragilexsyndrometreatedwithmetformintwocases
AT floratassone cognitiveandbehavioralimprovementinadultswithfragilexsyndrometreatedwithmetformintwocases
AT mariamtan cognitiveandbehavioralimprovementinadultswithfragilexsyndrometreatedwithmetformintwocases
AT randijhagerman cognitiveandbehavioralimprovementinadultswithfragilexsyndrometreatedwithmetformintwocases
AT andreaschneider cognitiveandbehavioralimprovementinadultswithfragilexsyndrometreatedwithmetformintwocases