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

Research output: Contribution to journalComment/debate

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 languageEnglish (US)
JournalNature Reviews Drug Discovery
Volume19
Issue number1
DOIs
StatePublished - Jan 1 2020

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Advisory Committees
Neuromuscular Diseases
Anniversaries and Special Events
Rare Diseases
Pharmaceutical Preparations
Therapeutics

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

Cite this

Wagner, K. R., De Luca, A., Caizergues, D., Dowling, J., Goemans, N., Gordish-Dressman, H., ... 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

A decade of optimizing drug development for rare neuromuscular disorders through TACT. / Wagner, Kathryn R.; De Luca, Annamaria; Caizergues, Didier; Dowling, James; Goemans, Nathalie; Gordish-Dressman, Heather; Grounds, Miranda D.; Kelly, Michael; Mayhew, Anna; McNally, Elizabeth M.; Zoetis, Tracey; Lee, Joanne; Turner, Cathy; Wells, Dominic J.; Csimma, Cristina; Straub, Volker.

In: Nature Reviews Drug Discovery, Vol. 19, No. 1, 01.01.2020.

Research output: Contribution to journalComment/debate

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
Wagner, Kathryn R. ; De Luca, Annamaria ; Caizergues, Didier ; Dowling, James ; Goemans, Nathalie ; Gordish-Dressman, Heather ; Grounds, Miranda D. ; Kelly, Michael ; Mayhew, Anna ; McNally, Elizabeth M. ; Zoetis, Tracey ; Lee, Joanne ; Turner, Cathy ; Wells, Dominic J. ; Csimma, Cristina ; Straub, Volker. / A decade of optimizing drug development for rare neuromuscular disorders through TACT. In: Nature Reviews Drug Discovery. 2020 ; Vol. 19, No. 1.
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