Trial reportMuscle & nerve2013
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.
Trial report in Muscle & nerve, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06290713 (Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy), which is not on this map. Cited by 154 papers, 4 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)
Who cites it
154 citing papers in PubMed, 4 syntheses or guidelines pooled it, 297 citations in OpenAlex.
- Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review.Paediatric drugs · 2024Pooled it
- Pooled it
- Reliability of Muscle Strength and Muscle Power Assessments Using Isokinetic Dynamometry in Neuromuscular Diseases: A Systematic Review.Physical therapy · 2022Pooled it
- The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review.Orphanet journal of rare diseases · 2017Pooled it
- Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.Annals of clinical and translational neurology · 2025Trial
- Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.Journal of comparative effectiveness research · 2025Trial
- Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.Journal of neurology · 2022Trial
- A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.Journal of neuromuscular diseases · 2022Trial
- Early phase 2 trial of TAS-205 in patients with Duchenne muscular dystrophy.Annals of clinical and translational neurology · 2020Trial
- Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.JAMA network open · 2019Trial
- Nusinersen improves walking distance and reduces fatigue in later-onset spinal muscular atrophy.Muscle & nerve · 2019Trial
- Quantifying disease activity in fatty-infiltrated skeletal muscle by IDEAL-CPMG in Duchenne muscular dystrophy.Neuromuscular disorders : NMD · 2016Trial
- Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.Annals of neurology · 2016Trial
- Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.Annals of neurology · 2016Trial
- Trial
- Upper limb evaluation and one-year follow up of non-ambulant patients with spinal muscular atrophy: an observational multicenter trial.PloS one · 2015Trial
- Trial
- A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Trial
- Ataluren treatment of patients with nonsense mutation dystrophinopathy.Muscle & nerve · 2014Trial
- The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.Muscle & nerve · 2013Trial
94 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
13 authors at 6 institutions in 2 countries.
Funding
Abstract
introductionDuchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized, double-blind, placebo-controlled trial of ataluren. Placebo arm data (N = 57) provided insight into the natural history of the 6-minute walk test (6MWT) and other endpoints.
methodsEvaluations performed every 6 weeks included the 6-minute walk distance (6MWD), timed function tests (TFTs), and quantitative strength using hand-held myometry.
resultsBaseline age (≥7 years), 6MWD, and selected TFT performance are strong predictors of decline in ambulation (Δ6MWD) and time to 10% worsening in 6MWD. A baseline 6MWD of <350 meters was associated with greater functional decline, and loss of ambulation was only seen in those with baseline 6MWD <325 meters. Only 1 of 42 (2.3%) subjects able to stand from supine lost ambulation.
conclusionFindings confirm the clinical meaningfulness of the 6MWD as the most accepted primary clinical endpoint in ambulatory DMD trials.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.