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Lookup NU author(s): Meredith JamesORCiD, Professor Michela GuglieriORCiD, Dr Anna Mayhew
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
© 2024 The AuthorsNorthStar Ambulatory Assessment (NSAA) total score (TS) is an ordinal scale to evaluate disease progression and treatment response in ambulatory Duchenne Muscular Dystrophy individuals. Clinical management according to standard of care could be enhanced by understanding how changes in the TS could inform standards of care. Here we describe the associated item performance patterns in the NorthStar Database for ranges of NSAA TS and its timed tests (10 m walk/run and rise from floor). We then compare these patterns depending on whether a participant is on an improving/stable (≤2-point loss in the prior year) or declining (>2-point loss in the prior year) trend. These TS and trends are subsequently linked and referenced to therapy standards of care. We included 761 participants from the UK NorthStar observational clinical database between 5 and 16 years, who were on steroids. Differences and trends in item ability, compensations, and times can suggest specific disease complications and lead towards anticipatory therapy recommendations. Families and therapists can benefit from using the TS and trend to guide therapy management.
Author(s): Stimpson G, James MK, Guglieri M, Wolfe A, Manzur A, Sarkozy A, Baranello G, Muntoni F, Mayhew A
Publication type: Article
Publication status: Published
Journal: European Journal of Paediatric Neurology
Year: 2024
Volume: 53
Pages: 123-130
Print publication date: 01/11/2024
Online publication date: 27/09/2024
Acceptance date: 11/09/2024
Date deposited: 11/11/2024
ISSN (print): 1090-3798
ISSN (electronic): 1532-2130
Publisher: W.B. Saunders Ltd
URL: https://doi.org/10.1016/j.ejpn.2024.09.004
DOI: 10.1016/j.ejpn.2024.09.004
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