A decade of optimizing drug development for rare neuromuscular disorders through TACT
- Wagner K. R.
- De Luca A.
- Caizergues D.
- Dowling J.
- Goemans N.
- Gordish-Dressman H.
- Grounds M. D.
- Kelly M.
- Mayhew A.
- McNally E. M.
- Zoetis T.
- Lee J.
- Turner C.
- Wells D. J.
- Csimma C.
- Straub V.
Publication date
January 2020
DOI Publisher
Springer Science and Business Media LLCAbstract
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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