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

IF 3.2 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2025-03-17 DOI:10.1177/22143602241313116

Anna G Mayhew, James Signorovitch, Michaela Johnson, Molly Frean, Susan J Ward, Nate Posner, Valeria Merla, Matthias Mahn, Georgia Stimpson, Michela Guglieri, Volker Straub, Robert Muni-Lofra, Adnan Manzur, Giovanni Baranello, Francesco Muntoni

引用次数: 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.

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来源期刊

Journal of neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.