ArticleJournal of neuromuscular diseases
Clinical Outcomes in Duchenne Muscular Dystrophy: A Study of 5345 Patients from the TREAT-NMD DMD Global Database.
Article in Journal of neuromuscular diseases. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02069756 (The Duchenne Registry), which is not on this map. Cited by 108 papers, 6 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.
The Duchenne Registry: An International, Patient-Report Registry for Individuals With Duchenne and Becker Muscular Dystrophy (Member of TREAT-NMD Neuromuscular Network)
Who cites it
108 citing papers in PubMed, 6 syntheses or guidelines pooled it.
- Predictors of cardiac disease in duchenne muscular dystrophy: a systematic review and evidence grading.Orphanet journal of rare diseases · 2024Pooled it
- Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis.Journal of neuromuscular diseases · 2024Pooled it
- Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading.Journal of neuromuscular diseases · 2024Pooled it
- Prognostic factors, disease course, and treatment efficacy in Duchenne muscular dystrophy: A systematic review and meta-analysis.Muscle & nerve · 2022Pooled it
- The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review.Orphanet journal of rare diseases · 2021Pooled it
- Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis.Orphanet journal of rare diseases · 2018Pooled it
- Preventing Cardiomyopathy in Duchenne Muscular Dystrophy: Long-Term Follow-Up of Patients in the Randomised, Placebo-Controlled Drug-Trial of Perindopril and Bisoprolol.European journal of neurology · 2025Trial
- Relationships betweenNeurology · 2019Trial
- Sociodemographic and Clinical Profile of Adult Males With Duchenne Muscular Dystrophy.Muscle & nerve · 2026Article
- The Effect of Corticosteroids on Pulmonary Function in a Large Cohort of Late Adolescents and Adults With Duchenne Muscular Dystrophy.Muscle & nerve · 2026Observational
- Effects of intermittent corticosteroids on scoliosis, bone density, and vertebral fractures in duchenne muscular dystrophy.Journal of neuromuscular diseases · 2026Article
- Beyond Coding Variants: RNA-Level Mechanisms in Human Disease and Precision Therapeutics.Genes · 2026Review
- Creatine/Creatinine Ratio and Myostatin as Biomarkers to Monitor Muscle Function in Duchenne Muscular Dystrophy Patients.Journal of cachexia, sarcopenia and muscle · 2026Observational
- Change in respiratory outcomes in adults with Duchenne muscular dystrophy in the era of corticosteroids.Journal of neuromuscular diseases · 2026Article
- Cardiac Medication Use in ACTION for Duchenne Muscular Dystrophy Cardiomyopathy.Pediatric cardiology · 2026Article
- Targeting Skeletal Muscle in Duchenne Muscular Dystrophy: Integrating in Silico and Experimental Approaches to Sodium-Glucose Cotransporter-2 Inhibition.The American journal of pathology · 2026Article
- Long-Term Safety and Efficacy of Systemic DT-DEC01 Cell Therapy in Non-Ambulatory Duchenne Muscular Dystrophy Patients: a 24-Month Clinical Evaluation.Stem cell reviews and reports · 2026Article
- Endocrinological complications of Duchenne muscular dystrophy and their subjective burden: observational study evaluating growth, puberty, and bone health.Journal of endocrinological investigation · 2026Observational
- From chromatin to proteostasis: multilevel gene regulation in Mendelian diseases.Frontiers in epigenetics and epigenomics · 2026Review
- State-of-the-Art on Model-Informed Drug Development Approaches for Pediatric Rare Diseases.CPT: pharmacometrics & systems pharmacology · 2025Review
48 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
64 authors.
Funding
Abstract
backgroundRecent short-term clinical trials in patients with Duchenne Muscular Dystrophy (DMD) have indicated greater disease variability in terms of progression than expected. In addition, as average life-expectancy increases, reliable data is required on clinical progression in the older DMD population.
objectiveTo determine the effects of corticosteroids on major clinical outcomes of DMD in a large multinational cohort of genetically confirmed DMD patients.
methodsIn this cross-sectional study we analysed clinical data from 5345 genetically confirmed DMD patients from 31 countries held within the TREAT-NMD global DMD database. For analysis patients were categorised by corticosteroid background and further stratified by age.
resultsLoss of ambulation in non-steroid treated patients was 10 years and in corticosteroid treated patients 13 years old (p = 0.0001). Corticosteroid treated patients were less likely to need scoliosis surgery (p < 0.001) or ventilatory support (p < 0.001) and there was a mild cardioprotective effect of corticosteroids in the patient population aged 20 years and older (p = 0.0035). Patients with a single deletion of exon 45 showed an increased survival in contrast to other single exon deletions.
conclusionsThis study provides data on clinical outcomes of DMD across many healthcare settings and including a sizeable cohort of older patients. Our data confirm the benefits of corticosteroid treatment on ambulation, need for scoliosis surgery, ventilation and, to a lesser extent, cardiomyopathy. This study underlines the importance of data collection via patient registries and the critical role of multi-centre collaboration in the rare disease field.
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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.