A patient-centered qualitative evaluation of meaningful change on the NSAA and PUL in Duchenne Muscular Dystrophy.

ABSTRACT:

Background

Duchenne Muscular Dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene. The North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL) are used to measure motor performance in ambulatory and non-ambulatory individuals, respectively. There is limited published qualitative evidence on what constitutes a meaningful change on either instrument. The aim of this study is to understand meaningful change in functional abilities as measured by the NSAA and PUL at different ability levels from the perspective of individuals with DMD, caregivers of individuals with DMD, and clinicians.

Methods

The study utilized a non-interventional, descriptive, cross-sectional qualitative design consisting of 69 semi-structured int

SUBMITTER: Gillman A 

PROVIDER: S-EPMC11915531 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature