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Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)

Willmann, R; Lee, J; Turner, C; Nagaraju, K; Aartsma-Rus, A; Wells, D J; Wagner, K R; Csimma, C; Straub, V; Grounds, M D; De Luca, A

Authors

R Willmann

J Lee

C Turner

K Nagaraju

A Aartsma-Rus

D J Wells

K R Wagner

C Csimma

V Straub

M D Grounds

A De Luca



Abstract

Clinical trials for rare neuromuscular diseases imply, among other investments, a high emotional burden for the whole disease community. Translation of data from preclinical studies to justify any clinical trial must be carefully pondered in order to minimize the risk of clinical trial withdrawal or failure. A rigorous distinction between proof-of-concept and preclinical efficacy studies using animal models is key to support the rationale of a clinical trial involving patients. This Review evaluates the experience accumulated by the TREAT-NMD Advisory Committee for Therapeutics, which provides detailed constructive feedback on clinical proposals for neuromuscular diseases submitted by researchers in both academia and industry, and emphasizes that a timely critical review of preclinical efficacy data from animal models, including biomarkers for specific diseases, combined with adherence to existing guidelines and standard protocols, can significantly help to de-risk clinical programs and prevent disappointments and costly engagement.

Citation

Willmann, R., Lee, J., Turner, C., Nagaraju, K., Aartsma-Rus, A., Wells, D. J., …De Luca, A. (2020). Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT). Disease Models and Mechanisms, 13(2), https://doi.org/10.1242/dmm.042903

Journal Article Type Review
Acceptance Date Feb 1, 2020
Publication Date Feb 7, 2020
Deposit Date Feb 18, 2020
Publicly Available Date Mar 10, 2020
Journal Disease Models and Mechanisms
Print ISSN 1754-8403
Electronic ISSN 1754-8411
Publisher The Company of Biologists
Peer Reviewed Peer Reviewed
Volume 13
Issue 2
DOI https://doi.org/10.1242/dmm.042903
Public URL https://rvc-repository.worktribe.com/output/1378740

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