Association of Gene Variant Classes With Motor Outcomes in a Drug Registration Clinical Trial Setting.

Background And Objectives: Duchenne muscular dystrophy (DMD) is caused by pathogenic variants of the gene, leading to the loss of dystrophin. Clinical variability in DMD can complicate interpretation of interventional data in clinical trials. One source of clinical variability is allelic heterogeneity (different pathogenic variants with different effects on dystrophin protein expression).