ArticleMuscle & nerve2014
Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy.
Article in Muscle & nerve, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03439670 (A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy), which is not on this map. Cited by 54 papers, 4 of them syntheses that pooled it.
What it found
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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.
A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)
Who cites it
54 citing papers in PubMed, 4 syntheses or guidelines pooled it, 102 citations in OpenAlex.
- Cardiac Phenotype-Genotype Associations in DMD/BMD: A Meta-Analysis and Systematic Review.Pediatric cardiology · 2021Pooled it
- Usefulness of speckle-tracking echocardiography for early detection in children with Duchenne muscular dystrophy: a meta-analysis and trial sequential analysis.Cardiovascular ultrasound · 2020Pooled it
- The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review.Orphanet journal of rare diseases · 2017Pooled it
- Guideline
- Cardiac Safety Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy with Up to 5 Years of Follow-up.Cardiology and therapy · 2026Article
- Influencing factors and early warning indicators for nonvertebral fractures in patients with Duchenne muscular dystrophy.JBMR plus · 2026Article
- Sodium-Glucose Co-Transporter 2 Inhibitors' Use in Muscular Dystrophy-Related Cardiomyopathy: Data from a Single-Center Experience.Journal of clinical medicine · 2026Article
- Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.Neurology and therapy · 2026Article
- Exploring the Role of Telemedicine in Duchenne Muscular Dystrophy: Benefits and Challenges.JMIR formative research · 2025Article
- Sinus Tachycardia and Unrelieved Wall Stress Precede Left Ventricular Systolic Dysfunction During Preclinical Cardiomyopathic Changes in Duchenne Muscular Dystrophy.Journal of cardiovascular development and disease · 2025Article
- The effects of glucocorticoids on cardiac function of patients with Duchenne muscular dystrophy: benefit or not?European journal of pediatrics · 2025Review
- Prophylactic Use of Cardiac Medications and Survival in Duchenne Muscular Dystrophy.Muscle & nerve · 2025Article
- Review
- Review
- Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.Journal of neuromuscular diseases · 2024Article
- The IAAM LTBP4 Haplotype is Protective Against Dystrophin-Deficient Cardiomyopathy.Journal of neuromuscular diseases · 2024Article
- Major Adverse Dystrophinopathy Events (MADE) Score as Marker of Cumulative Morbidity and Risk for Mortality in Boys with Duchenne Muscular Dystrophy.Progress in pediatric cardiology · 2023Article
- Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy.Frontiers in cell and developmental biology · 2023Article
- Current Practices in Treating Cardiomyopathy and Heart Failure in Duchenne Muscular Dystrophy (DMD): Understanding Care Practices in Order to Optimize DMD Heart Failure Through ACTION.Pediatric cardiology · 2022Article
- Tamoxifen treatment ameliorates contractile dysfunction of Duchenne muscular dystrophy stem cell-derived cardiomyocytes on bioengineered substrates.NPJ Regenerative medicine · 2022Article
Corrections and comments
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Authors and funding
7 authors at 4 institutions in 2 countries.
Funding
Abstract
introductionCardiomyopathy is a common cause of morbidity and death in patients with Duchenne muscular dystrophy (DMD).
methodsThis investigation was a cross-sectional cross-sectional analysis of clinical data from the multi-institutional Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study of 340 DMD patients aged 2-28 years. Cardiomyopathy was defined as shortening fraction (SF) <28% or ejection fraction (EF) <55%.
resultsTwo hundred thirty-one participants reported a prior clinical echocardiogram study, and 174 had data for SF or EF. The prevalence of cardiomyopathy was 27% (47 of 174), and it was associated significantly with age and clinical stage. The association of cardiomyopathy with age and clinical stage was not changed by glucocorticoid use as a covariate (P > 0.68). In patients with cardiomyopathy, 57% (27 of 47) reported not taking any cardiac medications. Cardiac medications were used in 12% (15 of 127) of patients without cardiomyopathy.
conclusionsWe found that echocardiograms were underutilized, and cardiomyopathy was undertreated in this DMD natural history cohort.
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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.