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Updated: Jan 19, 2026
Transaxillary First Rib Resection for Treatment of the Thoracic Outlet Syndrome
Published on: September 13, 2020
Metformin treatment in young children with fragile X syndrome
Hazel Maridith B Biag1,2, Laura A Potter1,2, Victoria Wilkins3
1Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, University of California Davis Medical Center, Sacramento, California.
Insights
Metformin shows promise for treating fragile X syndrome (FXS) in young children, improving language and behavior. Further controlled trials are needed to confirm these benefits in this critical developmental window.
Area of Science:
- Neuroscience
- Developmental Biology
- Pharmacology
Background:
- Fragile X syndrome (FXS) is a genetic disorder impacting cognitive and behavioral development.
- Metformin, a common diabetes drug, has shown potential in preclinical FXS models.
- Previous case studies suggest metformin may improve FXS-related symptoms in children and adults.
Observation:
- This study observed nine children aged 2-7 years with FXS treated with metformin.
- Behavioral and metabolic changes were monitored using parent reports and developmental testing.
Findings:
- Most children showed improvements in language development.
- Behavioral improvements included reduced lethargy and stereotypy.
Implications:
- These findings support a controlled trial of metformin for young children with FXS.
- Early intervention during critical brain development may enhance metformin's clinical benefits.
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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