Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy.

Nagy S, Schädelin S, Hafner P, Bonati U, Scherrer D, Ebi S, Schmidt S, Orsini AL, Bieri O, Fischer D.
Muscle Nerve. 2020. 61(1), 63-68.
In this retrospective study, the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD were analyzed. Clinical outcome measures included MFM.
MFM-D1 showed the highest sensitivity to change among all clinical tests in the analysis.

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Keywords: Duchenne muscular dystrophy; clinical outcome measure; longitudinal analysis; motor function measure; quantitative muscle MRI; sample size

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