Fragile X Syndrome and Targeted Treatments

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...

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Autores principales: Nattaporn Tassanakijpanich, Ana María Cabal-Herrera, Maria Jimena Salcedo-Arellano, Randi Jenssen Hagerman
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Lenguaje:EN
Publicado: Diponegoro University 2020
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Acceso en línea:https://doaj.org/article/e75efb3b52fc469bbf4c4b7a35bd2cbe
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spelling oai:doaj.org-article:e75efb3b52fc469bbf4c4b7a35bd2cbe2021-11-05T16:47:42ZFragile X Syndrome and Targeted Treatments2503-217810.14710/jbtr.v6i1.7321https://doaj.org/article/e75efb3b52fc469bbf4c4b7a35bd2cbe2020-04-01T00:00:00Zhttps://ejournal2.undip.ac.id/index.php/jbtr/article/view/7321https://doaj.org/toc/2503-2178Many 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.Nattaporn TassanakijpanichAna María Cabal-HerreraMaria Jimena Salcedo-ArellanoRandi Jenssen HagermanDiponegoro Universityarticlefragile x syndrome, fmr1, targeted treatmentMedicine (General)R5-920ENJournal of Biomedicine and Translational Research, Vol 6, Iss 1, Pp 23-33 (2020)
institution DOAJ
collection DOAJ
language EN
topic fragile x syndrome, fmr1, targeted treatment
Medicine (General)
R5-920
spellingShingle fragile x syndrome, fmr1, targeted treatment
Medicine (General)
R5-920
Nattaporn Tassanakijpanich
Ana María Cabal-Herrera
Maria Jimena Salcedo-Arellano
Randi Jenssen Hagerman
Fragile X Syndrome and Targeted Treatments
description 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.
format article
author Nattaporn Tassanakijpanich
Ana María Cabal-Herrera
Maria Jimena Salcedo-Arellano
Randi Jenssen Hagerman
author_facet Nattaporn Tassanakijpanich
Ana María Cabal-Herrera
Maria Jimena Salcedo-Arellano
Randi Jenssen Hagerman
author_sort Nattaporn Tassanakijpanich
title Fragile X Syndrome and Targeted Treatments
title_short Fragile X Syndrome and Targeted Treatments
title_full Fragile X Syndrome and Targeted Treatments
title_fullStr Fragile X Syndrome and Targeted Treatments
title_full_unstemmed Fragile X Syndrome and Targeted Treatments
title_sort fragile x syndrome and targeted treatments
publisher Diponegoro University
publishDate 2020
url https://doaj.org/article/e75efb3b52fc469bbf4c4b7a35bd2cbe
work_keys_str_mv AT nattaporntassanakijpanich fragilexsyndromeandtargetedtreatments
AT anamariacabalherrera fragilexsyndromeandtargetedtreatments
AT mariajimenasalcedoarellano fragilexsyndromeandtargetedtreatments
AT randijenssenhagerman fragilexsyndromeandtargetedtreatments
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