ePrints

Visualizing ambulatory performance by age and rates of decline among patients with Duchenne muscular dystrophy

Lookup NU author(s): Dr Anna Mayhew, Professor Michela GuglieriORCiD, Professor Volker StraubORCiD, Robert Muni Lofra

Downloads

• Published version [.pdf]

Licence

This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License (CC BY-NC 4.0).

Abstract

In Duchenne muscular dystrophy (DMD), age at symptom onset and rate of decline thereafter vary considerably. This study contrasted disease progression over time using the North Star Ambulatory Assessment (NSAA) in an overall sample of patients with DMD (mean age 7.1 years; baseline total NSAA score 22.2) with that of a centrally representative subgroup (mean age 6.9 years; NSAA score 24.0) defined according to median age at loss of ambulation. The average disease trajectory in the overall sample understated the more rapid rates of decline experienced by patients in the centrally representative subgroup.

Publication metadata

Author(s): Mayhew AG, Signorovitch J, Johnson M, Frean M, Ward SJ, Posner N, Merla V, Mahn Matthias, Stimpson G, Guglieri M, Straub S, Muni-Lofra R, Manzur A, Baranello G, Muntoni F

Publication type: Article

Publication status: Published

Journal: Journal of Neuromuscular Diseases

Year: 2025

Issue: ePub ahead of Print

Online publication date: 17/03/2025

Acceptance date: 22/12/2024

Date deposited: 21/05/2025

ISSN (print): 2214-3599

ISSN (electronic): 2214-3602

Publisher: Sage Publications, Inc.

URL: https://doi.org/10.1177/22143602241313116

DOI: 10.1177/22143602241313116

Data Access Statement: All relevant data are within the paper and its Supporting Information files. Individual data are available via data use agree- ment with The NorthStar Clinical Network database. Please contact Matthew Brooke at m.brooke@ucl.ac.uk. The authors did not receive special access privileges to the data

PubMed id: 40097910

Altmetrics

Funding

Share