Visualizing ambulatory performance by age and rates of decline among patients with Duchenne muscular dystrophy
- PMID: 40097910
- DOI: 10.1177/22143602241313116
Visualizing ambulatory performance by age and rates of decline among patients with Duchenne muscular dystrophy
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.
Keywords: Duchenne muscular dystrophy; North Star Ambulatory Assessment; ambulation; disease progression; visualization.
Conflict of interest statement
Declaration of conflicting interestsThe authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: AGM has served on medical/scientific advisory boards for Regenxbio, Sarepta, Biogen and Roche; and has received fees for consulting and training services for Biogen, Roche, Novartis, Biohaven, PTC, Sarepta, Italfarmaco, Dyne, Pfizer, Summit, Catabasis, Capricor, Santhera, Vision, Lysogene, Modis, Amicus, Taysha, Antisense, Analysis Group, MDUK and DUK. JS, MJ, and MF are employees of Analysis Group, Inc., a consulting company that has provided paid consulting services to Pfizer Inc., which funded this study. SJW has received consultancy fees from CureDuchenne, PPMD, Charley's Fund and Analysis Group. NP and VM are employees of Pfizer Inc. and own stock/stock options. MM was an employee of Pfizer Inc. at the time this study was conducted and held stock/stock options. GS has no conflicts of interest to disclose. MG has served on medical/scientific advisory boards for Pfizer, Dyne, NS Pharma, Antisense; has received research grants from PTC Therapeutics, Sarepta, Edgewise and was on the speaker's bureau for Italfarmaco, Sarepta, Pfizer, Novartis, Roche. VS is an Editorial Board Member of this journal, but was not involved in the peer-review process nor had access to any information regarding its peer-review. In addition, VS has received research grants from Sarepta Therapeutics and Sanofi, consultancy fees from Astellas Gene Therapies, Biogen, Edgewise Therapeutics, Ipsen, Kate Therapeutics, ML Bio Solutions, Novartis Gene Therapies, PepGen, Roche, Sanofi, Sarepta Therapeutics, Vertex Pharmaceuticals, and Wave Therapeutics, and was on the speaker's bureau for Pfizer, Roche, Sanofi, and Sarepta Therapeutics. RML has received research grants from Roche, consultancy fees from Roche, Biogen and Novartis, and was on the speaker's bureau for Biogen and Roche. AM has no conflicts of interest to disclose. GB is a principal investigator of clinical trials sponsored by Roche, Novartis, Sarepta, Pfizer, NS Pharma, Reveragen, Scholar Rock, and has received speaker and/or consulting fees from Sarepta, PTC Therapeutics, Pfizer, Biogen, Novartis Gene Therapies, Inc. (AveXis), and Roche, and grants from Sarepta, Roche and Novartis Gene Therapies. FM reports consultancy fees from Sarepta, Pfizer, Dyne Therapeutics, Kate Therapeutics, PTC Therapeutics and Roche, and research grants from Sarepta Therapeutics. FM has also participated in educational symposia organised by Roche, Sarepta and PTC Therapeutics.
