Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial arms
Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial arms The design of clinical trials and evaluations of treatment efficacy in DMD require an understanding of the meaningfulness of changes in functional measures. An important concept is the minimal detectable change (MDC), i.e., the minimal magnitude of measured change indicative of true changes due to acquisition or loss of motor function related to the disease, rather...