Human Molecular Genetics and Stem Cell Laboratory, Department of Human Genetics and Molecular Biology, Bharathiar University, Coimbatore, Tamil Nadu, 641046, India.
Integrated BS-MS, Indian Institute of Science Education and Research, Kolkata, India.
J Autism Dev Disord. 2020 Aug;50(8):2740-2764. doi: 10.1007/s10803-020-04381-y.
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.
过去几十年，有关雷特综合征（RTT）的药物研究大幅增加。本综述旨在提供涉及 RTT 的药物从实验室到临床的研究的主要数据。通过在 PUBMED、MEDLINE 和 Google Scholar 中搜索、国际、国家和地区临床试验注册机构以及制药公司，使用“Rett 综合征治疗和/或药物或化合物或分子”等关键词，进行了全面的文献综述。有 70 种药物在非临床（N=65 种基于动物/细胞系的研究；N=5 种基于 iPSC 的研究）和临床试验（N=34）中被研究用于改善 RTT 的症状。尽管在临床和非临床研究方面都取得了良好的进展，但这些药物都没有进入 III/IV 期，也没有一种被作为 RTT 的治疗药物上市。
