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

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

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...

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Bibliographic Details
Main Authors: Raffaella Willmann, Joanne Lee, Cathy Turner, Kanneboyina Nagaraju, Annemieke Aartsma-Rus, Dominic J. Wells, Kathryn R. Wagner, Cristina Csimma, Volker Straub, Miranda D. Grounds, Annamaria De Luca, James Dowling, Maaike van Putten
Format: Article
Language:English
Published: The Company of Biologists 2020-02-01
Series:Disease Models & Mechanisms
Subjects:
animal models
preclinical
guidelines
efficacy studies
clinical trial
standard protocols
neuromuscular
Online Access:http://dmm.biologists.org/content/13/2/dmm042903

Internet

http://dmm.biologists.org/content/13/2/dmm042903

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