Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study

Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study

Evaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the ‘meaningfulness’ of changes in functional measures. We estimated the minimal detectable change (MDC) for selected motor function...

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Bibliographic Details
Main Authors:	Muntoni, F, Signorovitch, J, Sajeev, G, Done, N, Yao, Z, Goemans, N, McDonald, C, Mercuri, E, Niks, EH, Wong, B, Vandenborne, K, Straub, V, de Groot, IJM, Tian, C, Manzur, A, Dieye, I, Lane, H, Ward, SJ, Servais, L, PRO-DMD-01 study investigators, Association Française contre les Myopathies, The UK NorthStar Clinical Network, ImagingDMD investigators, cTAP
Format:	Journal article
Language:	English
Published:	Public Library of Science 2024

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