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

doi:10.1038/d41573-019-00199-1

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

Kennedy Krieger Institute

Research output: Contribution to journal › Comment/debate › peer-review

2 Scopus citations

Abstract

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.

Original language	English (US)
Journal	Nature Reviews Drug Discovery
Volume	19
Issue number	1
DOIs	https://doi.org/10.1038/d41573-019-00199-1
State	Published - Jan 1 2020

ASJC Scopus subject areas

Pharmacology
Drug Discovery

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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. (2020). A decade of optimizing drug development for rare neuromuscular disorders through TACT. Nature Reviews Drug Discovery, 19(1). https://doi.org/10.1038/d41573-019-00199-1

Wagner, KR, De Luca, A, Caizergues, D, Dowling, J, Goemans, N, Gordish-Dressman, H, Grounds, MD, Kelly, M, Mayhew, A, McNally, EM, Zoetis, T, Lee, J, Turner, C, Wells, DJ, Csimma, C & Straub, V 2020, 'A decade of optimizing drug development for rare neuromuscular disorders through TACT', Nature Reviews Drug Discovery, vol. 19, no. 1. https://doi.org/10.1038/d41573-019-00199-1

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AU - Goemans, Nathalie

AU - Gordish-Dressman, Heather

AU - Grounds, Miranda D.

AU - Kelly, Michael

AU - Mayhew, Anna

AU - McNally, Elizabeth M.

AU - Zoetis, Tracey

AU - Lee, Joanne

AU - Turner, Cathy

AU - Wells, Dominic J.

AU - Csimma, Cristina

AU - Straub, Volker

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

Abstract

ASJC Scopus subject areas

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