Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy.

Background And Objectives: Understanding the reliability of outcomes used in clinical care and trials is important to delineate intervention-induced change from random variability. Reliability, sensitivity, and clinical meaningfulness of change are all key aspects of choosing the most appropriate outcome measure for a clinical trial. Common outcome measures to monitor progression and treatment effect in Duchenne muscular dystrophy (DMD) include measures of strength (myometry) and motor function tests: stand from supine velocity (STANDV), North Star Ambulatory Assessment (NSAA), 6-minute walk distance (6MWD), 10-m run/walk (RWV), and 4-stair climb velocity (CLIMBV).