Metformin treatment in young children with fragile X syndrome
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 metf...
| Main Authors: | , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Wiley
2019-11-01
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| Series: | Molecular Genetics & Genomic Medicine |
| Subjects: | |
| Online Access: | https://doi.org/10.1002/mgg3.956 |
| _version_ | 1797296143339618304 |
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| author | Hazel Maridith B. Biag Laura A. Potter Victoria Wilkins Sumra Afzal Alexis Rosvall Maria Jimena Salcedo‐Arellano Akash Rajaratnam Ramiro Manzano‐Nunez Andrea Schneider Flora Tassone Susan M. Rivera Randi J. Hagerman |
| author_facet | Hazel Maridith B. Biag Laura A. Potter Victoria Wilkins Sumra Afzal Alexis Rosvall Maria Jimena Salcedo‐Arellano Akash Rajaratnam Ramiro Manzano‐Nunez Andrea Schneider Flora Tassone Susan M. Rivera Randi J. Hagerman |
| author_sort | Hazel Maridith B. Biag |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-07T21:59:07Z |
| format | Article |
| id | doaj.art-7178a3672dca48a98ee5a540a0511c15 |
| institution | Directory Open Access Journal |
| issn | 2324-9269 |
| language | English |
| last_indexed | 2024-03-07T21:59:07Z |
| publishDate | 2019-11-01 |
| publisher | Wiley |
| record_format | Article |
| series | Molecular Genetics & Genomic Medicine |
| spelling | doaj.art-7178a3672dca48a98ee5a540a0511c152024-02-24T07:01:11ZengWileyMolecular Genetics & Genomic Medicine2324-92692019-11-01711n/an/a10.1002/mgg3.956Metformin treatment in young children with fragile X syndromeHazel Maridith B. Biag0Laura A. Potter1Victoria Wilkins2Sumra Afzal3Alexis Rosvall4Maria Jimena Salcedo‐Arellano5Akash Rajaratnam6Ramiro Manzano‐Nunez7Andrea Schneider8Flora Tassone9Susan M. Rivera10Randi J. Hagerman11Medical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaDepartment of Pediatric Inpatient Medicine University of Utah and Primary Children’s Hospital Salt Lake City, UtahMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaUniversity of California Davis School of Medicine Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaFundación Valle del Lili Clinical Research Center Cali ColombiaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaMedical Investigation of Neurodevelopmental Disorders (MIND) Institute University of California Davis Medical Center Sacramento CaliforniaAbstract 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.https://doi.org/10.1002/mgg3.956FMR1fragile X syndromemetformintargeted treatmentstranslational medicine |
| spellingShingle | Hazel Maridith B. Biag Laura A. Potter Victoria Wilkins Sumra Afzal Alexis Rosvall Maria Jimena Salcedo‐Arellano Akash Rajaratnam Ramiro Manzano‐Nunez Andrea Schneider Flora Tassone Susan M. Rivera Randi J. Hagerman Metformin treatment in young children with fragile X syndrome Molecular Genetics & Genomic Medicine FMR1 fragile X syndrome metformin targeted treatments translational medicine |
| title | Metformin treatment in young children with fragile X syndrome |
| title_full | Metformin treatment in young children with fragile X syndrome |
| title_fullStr | Metformin treatment in young children with fragile X syndrome |
| title_full_unstemmed | Metformin treatment in young children with fragile X syndrome |
| title_short | Metformin treatment in young children with fragile X syndrome |
| title_sort | metformin treatment in young children with fragile x syndrome |
| topic | FMR1 fragile X syndrome metformin targeted treatments translational medicine |
| url | https://doi.org/10.1002/mgg3.956 |
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