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Maria Jimena Salcedo-Arellano
UC Davis MIND Institute
Biochemistry, Genetics & Molecular Biology Medicine & Pharmacology

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Fragile X Syndrome and Targeted Treatments Nattaporn Tassanakijpanich; Ana María Cabal-Herrera; Maria Jimena Salcedo-Arellano; Randi Jenssen Hagerman
Journal of Biomedicine and Translational Research Vol 6, No 1 (2020): April 2020
Publisher : Faculty of Medicine, Diponegoro University

Show Abstract | Download Original | Original Source | Check in Google Scholar | DOI: 10.14710/jbtr.v6i1.7321

Abstract

Many targeted treatment studies have been carried out in individuals with Fragile X Syndrome (FXS) guided by animal studies from the Fragile X Mental Retardation 1 (FMR1) knock out (KO) mice and the fragile X Drosophila studies. Here we review the many medications that have been studied in patients with FXS and some of these medications are available for clinical use by wise clinicians. Other medications are not currently available by prescription because they are not approved by the FDA. No medication has received specific approval for treatment of FXS, although some have shown benefit from clinical studies. There is much to be done in the treatment of those with FXS and this report describes those pharmacological treatments that target the neurobiological mechanisms that are dysregulated by the lack of the Fragile X Protein (FMRP) in those with FXS.
Co-Authors Ana María Cabal-Herrera Nattaporn Tassanakijpanich Randi Jenssen Hagerman