Brief Report: Acamprosate in Fragile X Syndrome
Abstract
Glutamatergic dysfunction is implicated in the pathophysiology of fragile X syndrome (FXS). We report on the first trial of acamprosate, a drug with putative mGluR5 antagonism, in three adults with FXS and autism. Medical records describing open-label treatment with acamprosate in 3 patients with FXS and a comorbid diagnosis of autistic disorder were reviewed. In all three patients, acamprosate was associated with improved linguistic communication. Three patients received acamprosate over a mean 21.3 weeks of treatment. All patients showed global clinical benefit as rated with the Clinical Global Impressions-Improvement scale. Marked communication improvement was unexpected and has potential implications for the treatment of FXS, as well as idiopathic autism.
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Acamprosate blocks NMDA receptors and activates GABA A receptors; in three young adult patients with fragile X syndrome it produced global benefit and marked communication improvement.
“Acamprosate, a molecule used to maintain abstinence from alcohol, is able to block NMDA-R as well as activate GABA A -R and was recently tested in three young adult patients with FXS, who showed global benefit (as rated by the CGI-I scale) and marked communication improvement (115).”
From Fragile X syndrome: causes, diagnosis, mechanisms, and therapeutics (2012) · cites this paper as [115]
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- Authors
- Erickson, C. A., Mullett, J. E., & McDougle, C. J.
- Affiliations
- Riley Hospital for Children (US)
- Corresponding author
- Erickson, Craig A.
- Venue
- Journal of Autism and Developmental Disorders
- Published
- 2010-03-06
- Volume
- 40
- Issue
- 11
- Pages
- 1412-1416
- ISSN
- 0162-3257 (ISSN-L)
- 1573-3432
- OpenAlex
- W2035030950
- Cited by
- 67
- References
- 32
- Citations by year
- 2023: 1 · 2022: 3 · 2021: 2 · 2019: 3 · 2018: 6 · 2017: 6 · 2016: 5 · 2015: 7 · 2014: 6 · 2013: 9 · 2012: 9
- FWCI
- 5.04
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Journal of Autism and Developmental Disorders (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institute of Mental Health (R01MH072964)
- National Center for Research Resources (K12 UL1 RR025761, UL1RR025761)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Neuroscience and Neuropharmacology Research
- Keywords
- acamprosate, fragile X syndrome, autism spectrum disorder, glutamatergic dysfunction, linguistic communication
- MeSH terms
- Acamprosate, Adolescent, Autistic Disorder, Communication, Fragile X Syndrome, Humans, Male, Taurine, Verbal Behavior, Comorbidity, Treatment Outcome, Receptors, Metabotropic Glutamate, Young Adult, Receptor, Metabotropic Glutamate 5
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