Development of a patient‐reported outcome measure for upper limb function in Duchenne muscular dystrophy: <scp>DMD</scp> Upper Limb <scp>PROM</scp>

Abstract

A DMD-specific upper limb PROM was developed on the basis of clinical relevance and psychometric robustness. Its main purpose is to document the patient self-reported natural history of DMD and assess the efficacy of interventions.