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A decade of optimizing drug development for rare neuromuscular disorders through TACT.
Nature Reviews Drug Discovery ( IF 122.7 ) Pub Date : 2020-01-01 , DOI: 10.1038/d41573-019-00199-1
Kathryn R Wagner , Annamaria De Luca , Didier Caizergues , James Dowling , Nathalie Goemans , Heather Gordish-Dressman , Miranda D Grounds , Michael Kelly , Anna Mayhew , Elizabeth M McNally , Tracey Zoetis , Joanne Lee , Cathy Turner , Dominic J Wells , Cristina Csimma , Volker Straub

This year marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare neuromuscular diseases and identifies pitfalls. Here, we discuss the experience with TACT based on its reviews of more than 50 applications and its potential as a model for other rare disorders.

中文翻译:

通过TACT为罕见的神经肌肉疾病优化药物开发的十年。

今年是TREAT-NMD治疗学咨询委员会(TACT)成立十周年,该委员会由多学科专家组成,负责评估罕见神经肌肉疾病的药物开发计划并确定陷阱。在这里,我们将基于TACT对50多种应用的评论以及其作为其他罕见疾病模型的潜力,来讨论TACT的经验。
更新日期:2019-11-19
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