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

ABSTRACT: 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 measures in ambulatory DMD, i.e., the minimal degree of measured change needed to be confident that true underlying change has occurred rather than transient variation or measurement error. MDC estimates were compared across multiple data sources, representing >1000 DMD patients in clinical trials and real-world clinical practice settings. Included patients were ambulatory, aged ≥4 to <18 years and receiving steroids. Minimal clinically important differences (MCIDs) for worsening were also estim

SUBMITTER: Muntoni F 

PROVIDER: S-EPMC11236155 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature