A decade of optimizing drug development for rare neuromuscular disorders through TACT

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

NATURE REVIEWS DRUG DISCOVERY（2019）

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摘要

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.

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