Patient-centered benefit-risk assessment in duchenne muscular dystrophy.


INTRODUCTION This study quantified caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregiver and patient differences were also explored. METHODS A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the… (More)
DOI: 10.1002/mus.25411


Cite this paper

@article{Hollin2017PatientcenteredBA, title={Patient-centered benefit-risk assessment in duchenne muscular dystrophy.}, author={Ilene L Hollin and Holly Peay and Susan D. Apkon and John F. P. Bridges}, journal={Muscle & nerve}, year={2017}, volume={55 5}, pages={626-634} }