ArticleClinical investigation2011
Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings.
Article in Clinical investigation, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 77 papers, 1 of them a synthesis 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
77 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Effect of Muscular Exercise on Patients With Muscular Dystrophy: A Systematic Review and Meta-Analysis of the Literature.Frontiers in neurology · 2020Pooled it
- Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.Journal of neuromuscular diseases · 2022Trial
- A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial.Journal of neuromuscular diseases · 2021Trial
- Trial
- Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.Muscle & nerve · 2018Trial
- Trial
- Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.Annals of neurology · 2016Trial
- Trial
- Functional assessments used in routine care for duchenne muscular dystrophy in the United States: A survey of certified duchenne care centers.Journal of neuromuscular diseases · 2026Article
- Combination S100A1 and ARC gene therapy as a treatment for Duchenne muscular dystrophy cardiomyopathy.JCI insight · 2026Article
- Upper limb progression in Duchenne muscular dystrophy: Insights from a 36-month longitudinal study using the PUL 20.Journal of neuromuscular diseases · 2026Article
- Quantitative MRI Findings and Their Relationship to Muscle Histopathology and Ambulatory Clinical Function in Duchenne Muscular Dystrophy.Journal of cachexia, sarcopenia and muscle · 2026Article
- Quantitative Muscle Ultrasound: A Non-Invasive Biomarker for Monitoring Duchenne Muscular Dystrophy.Muscle & nerve · 2025Observational
- Exploring the Role of Telemedicine in Duchenne Muscular Dystrophy: Benefits and Challenges.JMIR formative research · 2025Article
- Article
- Physical activity and cardiac function in patients with Duchenne muscular dystrophy.Cardiology in the young · 2025Article
- Psychometric Assessment of the Rett Syndrome Caregiver Assessment of Symptom Severity (RCASS).Journal of autism and developmental disorders · 2025Article
- "If you cannot measure it, you cannot improve it". Outcome measures in Duchenne Muscular Dystrophy: current and future perspectives.Acta neurologica Belgica · 2025Review
- Article
- RNA mis-splicing in children with congenital myotonic dystrophy is associated with physical function.Annals of clinical and translational neurology · 2024Article
17 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
In June 2010, 25 representatives from Europe and the US met in Washington, DC, USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the context of clinical trial design and analysis. The workshop was organized in response to a September 2009 European Medicines Agency meeting where a clear directive was given that an international consensus needs to be developed that provides a foundation for age-appropriate clinical outcome measures for use in clinical trials of emerging therapeutics for DMD. Data were presented from eight multicenter longitudinal datasets, representing nearly 1900 patients over a 20-year time period. This experience confirmed the feasibility of repeated evaluations performed at multiple sites and addressed several core issues in drug development for DMD, such as the 'new' natural history in the steroidera, reliability and sensitivity of specific outcome measures, as well as disease staging and patient selection. These data form a valuable asset for academic investigators, pharmaceutical sponsors and regulatory agencies involved in DMD therapeutics. The group remains committed working together on a number of collaborative goals to support the therapeutics development effort in this orphan disease and to make these data available to stakeholders working in the field.
Identifiers
What OpenQuestion holds
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.