Abstract
The progression of DMD is characterized by loss of ambulation (LOA) and upper limb function, cardiorespiratory impairment, and early mortality. While much attention is focused on LOA, the impact of other aspects of progression on HRQoL is less clear. Understanding patient perspectives on these aspects is important to provide context to clinical health states in DMD, and link changes in DMD symptoms to patient experiences. The objective of this study was to obtain insights on how key health state transitions affect HRQoL among individuals with DMD.
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