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

Reprinted from Orphanet Journal of Rare DiseasesApril 20, 2023By: James E. Valentine Download PDF

Patient-centered research has emerged as critically important for understanding the impact of treatments on key stakeholders. 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).

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