Quantitative muscle MRI biomarkers in Duchenne muscular dystrophy: cross-sectional correlations with age and functional tests.

Lay abstract Clinical trials in Duchenne muscular dystrophy have proven to be challenging, due in part to the lack of robust biomarkers that are sensitive to detecting disease progression. While physical function, such as walking or climbing stairs, will be critical to demonstrating the long-term efficacy of a therapeutic, MRI may be a more objective approach that could detect subtle changes in disease status and offer earlier signals of clinical efficacy. In this study, researchers used baseline data from a Phase II clinical study (NCT02310763) to evaluate the relationship between measures of physical function and quantitative MRI assessments. They found that many MRI measures were moderately correlated with physical function, and muscle volume measurements were correlated with measures of strength. This study supports the use of MRI as a biomarker in Duchenne muscular dystrophy clinical trials.