Clinical importance of changes in magnetic resonance biomarkers for Duchenne muscular dystrophy.

ABSTRACT:

Objective

Magnetic resonance (MR) measures of muscle quality are highly sensitive to disease progression and predictive of meaningful functional milestones in Duchenne muscular dystrophy (DMD). This investigation aimed to establish the reproducibility, responsiveness to disease progression, and minimum clinically important difference (MCID) for multiple MR biomarkers at different disease stages in DMD using a large natural history dataset.

Methods

Longitudinal MR imaging and spectroscopy outcomes and ambulatory function were measured in 180 individuals with DMD at three sites, including repeated measurements on two separate days (within 1 week) in 111 participants. These data were used to calculate day-to-day reproducibility, responsiveness (standardized response mean, SRM),

SUBMITTER: Willcocks RJ 

PROVIDER: S-EPMC10791017 | biostudies-literature | 2024 Jan

REPOSITORIES: biostudies-literature