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Gepubliceerd in: Journal of Autism and Developmental Disorders 8/2020

03-02-2020 | Original Paper

Drug Studies on Rett Syndrome: From Bench to Bedside

Auteurs: Mohan Gomathi, Subramanian Padmapriya, Vellingiri Balachandar

Gepubliceerd in: Journal of Autism and Developmental Disorders | Uitgave 8/2020

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Abstract

Drug studies on Rett syndrome (RTT) have drastically increased over the past few decades. This review aims to provide master data on bench-to-bedside drug studies involving RTT. A comprehensive literature review was performed by searching in PUBMED, MEDLINE and Google Scholar, international, national and regional clinical trial registries and pharmaceutical companies using the keywords “Rett syndrome treatment and/or drug or compound or molecule”. Seventy drugs were investigated in non-clinical (N = 65 animal/cell line-based studies; N = 5 iPSC-based study) and clinical trials (N = 34) for ameliorating the symptoms of RTT. Though there is good progress in both clinical and non-clinical studies, none of these drugs entered phase III/IV for being launched as a therapeutic agent for RTT.
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Metagegevens
Titel
Drug Studies on Rett Syndrome: From Bench to Bedside
Auteurs
Mohan Gomathi
Subramanian Padmapriya
Vellingiri Balachandar
Publicatiedatum
03-02-2020
Uitgeverij
Springer US
Gepubliceerd in
Journal of Autism and Developmental Disorders / Uitgave 8/2020
Print ISSN: 0162-3257
Elektronisch ISSN: 1573-3432
DOI
https://doi.org/10.1007/s10803-020-04381-y

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