ArticleCPT: pharmacometrics & systems pharmacology2017
Developing a Natural History Progression Model for Duchenne Muscular Dystrophy Using the Six-Minute Walk Test.
Article in CPT: pharmacometrics & systems pharmacology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review.Paediatric drugs · 2024Pooled it
- Fully personalized modelling of Duchenne Muscular Dystrophy ambulation.Philosophical transactions. Series A, Mathematical, physical, and engineering sciences · 2025Article
- Feasibility and usability of a wearable sensor system for gait assessment in children with neuromuscular diseases.Frontiers in rehabilitation sciences · 2025Article
- Appendicular lean mass index changes in patients with Duchenne muscular dystrophy and Becker muscular dystrophy.Journal of cachexia, sarcopenia and muscle · 2023Article
- Efficacy and safety of hydrokinesitherapy in patients with dystrophinopathy.Frontiers in neurology · 2023Article
- Good response to the late treatment with ataluren in a boy with Duchenne muscular dystrophy: could the previous mild course of the disease have affected the outcome?Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2022Article
- Latent process model of the 6-minute walk test in Duchenne muscular dystrophy : A Bayesian approach to quantifying rare disease progression.Journal of pharmacokinetics and pharmacodynamics · 2020Article
- Effectiveness of pharmacological treatments in Duchenne muscular dystrophy: a protocol for a systematic review and meta-analysis.BMJ open · 2019Article
- Empirical and Computational Comparison of Alternative Therapeutic Exon Skip Repairs for Duchenne Muscular Dystrophy.Biochemistry · 2019Article
- Can simple and low-cost motor function assessments help in the diagnostic suspicion of Duchenne muscular dystrophy?Jornal de pediatriaArticle
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Authors and funding
4 authors.
Funding
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Abstract
The 6-minute walk test (6MWT) is used as a clinical endpoint to evaluate drug efficacy in Duchenne Muscular Dystrophy (DMD) trials. A model was developed using digitized 6MWT data that estimated two slopes and two intercepts to characterize 6MWT improvement during development and 6MWT decline. Mean baseline 6MWT was 362 (±87) meters. The model predicted an improvement at a rate of 20 meters/year (95% confidence interval (CI) = 9.4-30) up until 10 years old (95% CI = 6.78-13.1), and then a decline at a rate of 85 meters/year (95% CI = 72-98). Interpatient slope variability for improvement and decline were similar at 21.9 percentage of coefficient of variation (%CV) and 23.3%CV, respectively. Model simulations using age demographics from a previous DMD natural history study could reasonably predict the trend in improvement and decline in the 6MWT. This model can be used to quantitate individual patient trajectories, identify prognostic factors for disease progression, and evaluate drug effect.
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