Population
81 well-defined Facioscapulohumeral muscular dystrophy (FSHD) patients
Design
Cohort
Follow-up
up to 3 years
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Supports MVICT/MMT as progression measures in FSHD trials; extends observational data for sample size estimates.
This study establishes reliable measures of disease progression for FSHD clinical trials and provides sample size guidelines for future studies.
A 1997 study studied this question.
Synapse has enriched 4 closely related papers on similar clinical questions. Consider them for comparative context: