MRI biomarker assessment of Duchenne muscular dystrophy disease progression: a 12-month longitudinal study

Motivation: Duchenne muscular dystrophy (DMD) leads to progressive muscle degeneration, yet reliable biomarkers to monitor disease progression over time are limited. Goal(s): To evaluate DMD disease progression using qMRI and compare the responsiveness of imaging indicators with clinical function scales. Approach: Patients underwent gluteal muscle MRI for fat fraction (FF) and T1 relaxation time. Motor function was evaluated using the North Star Ambulatory Assessment (NSAA) and timed functional tests, with standardized response mean (SRM) measuring disease progression responsiveness. Results: The overall SRM of MRI biomarkers is higher than that of the clinical function scales, and their sensitivity indicators vary with the disease stages. Impact: The high sensitivity of MRI biomarkers to subtle muscle changes enables early detection of pathology, making it a valuable tool for tracking disease progression from subclinical to symptomatic stages.