Aims/hypothesis It is generally accepted that hypoglycaemia can negatively impact the
quality of life (QoL) of people living with diabetes. However, the suitability of patient-reported …

Introduction Duchenne muscular dystrophy is a rare, progressive, life-limiting genetic
neuromuscular condition that significantly impacts the quality of life of informal caregivers …

Objective Based on concerns about existing patient-reported outcome measures (PROMs)
for assessing quality of life (QoL) in Duchenne muscular dystrophy (DMD), we describe the …

Purpose Duchenne muscular dystrophy (DMD) is a rare x-linked neuromuscular condition
predominantly affecting boys and men. There is a paucity of research qualitatively detailing …

Objectives This study generates a preference-based measure for capturing the quality of life
of people with Duchenne muscular dystrophy (DMD) from a new measure of quality of life …

Background Duchenne muscular dystrophy is a rare degenerative neuromuscular disorder
with pediatric onset. Recent approvals in Duchenne have placed attention on the economic …

Purpose Cardiac disease results in significant morbidity and mortality in patients with
muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) …

Abstract Objectives Health-related Quality of Life (HRQoL) instruments for cardiovascular
diseases (CVD) have been commonly used to measure important patient-reported outcomes …

Background There is no widely accepted framework to guide the development of condition-
specific preference-based instruments (CSPBIs) that includes both de novo and from …

Background Data on health state utility in Duchenne muscular dystrophy (DMD) are few.
This study estimated mean utility values by age, ambulatory status and over time, and …