Drug Studies on Rett Syndrome: From Bench to Bedside / Mohan GOMATHI in Journal of Autism and Developmental Disorders, 50-8 (August 2020)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 50-8 (August 2020) . - p.2740-2764 Titre : Drug Studies on Rett Syndrome: From Bench to Bedside Type de document : texte imprimé Auteurs : Mohan GOMATHI, Auteur ; Subramanian PADMAPRIYA, Auteur ; Vellingiri BALACHANDAR, Auteur Article en page(s) : p.2740-2764 Langues : Anglais (eng) Mots-clés : Clinical trials  Drug discovery  MECP2 knockout mice  Non-clinical studies  Rett syndrome  iPSC Index. décimale : PER Périodiques Résumé : 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. En ligne : http://dx.doi.org/10.1007/s10803-020-04381-y Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=428 [article] Drug Studies on Rett Syndrome: From Bench to Bedside [texte imprimé] / Mohan GOMATHI, Auteur ; Subramanian PADMAPRIYA, Auteur ; Vellingiri BALACHANDAR, Auteur . - p.2740-2764. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 50-8 (August 2020) . - p.2740-2764 Mots-clés : Clinical trials  Drug discovery  MECP2 knockout mice  Non-clinical studies  Rett syndrome  iPSC Index. décimale : PER Périodiques Résumé : 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. En ligne : http://dx.doi.org/10.1007/s10803-020-04381-y Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=428

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