DMD & BMD – CLINICAL: P.57 Comparison of North Star Ambulatory Assessment scores in siblings with Duchenne muscular dystrophy

Abstract

Early diagnosis is important to optimise management and prolong function in patients with Duchenne muscular dystrophy (DMD). There is little research comparing functional outcomes of siblings diagnosed at different ages. This study aims to compare peak North Star Ambulatory Assessment (NSAA) scores in siblings in whom the optimal standards of care were implemented at different ages. NSAA data from 24 siblings (48 patients) were analysed from their first NSAA assessment at Great Ormond Street Hospital, including all scores until January 2020.