Toward patient-centered treatment goals for duchenne muscular dystrophy: insights from the “Your Voice” study

Background: Patient-centered research has emerged as critically important for understanding the impact of treatments on patients. The subjective experience of quality of life (QOL) is increasingly recognized as fundamental to delineating treatment goals. The present study utilized content analysis of qualitative data and quantitative analysis to highlight important domains of disease burden and underlying reasons for their importance, and to characterize goals for new treatments for Duchenne Muscular Dystrophy (DMD).