ArticleJournal of neuromuscular diseases2016
European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent Differences.
Article in Journal of neuromuscular diseases, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers, 7 of them syntheses that pooled it.
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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.
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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.
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Who cites it
34 citing papers in PubMed, 7 syntheses or guidelines pooled it, 96 citations in OpenAlex.
- Improving survival in Duchenne muscular dystrophy across eras: a systematic review and cumulative meta-analysis.Journal of neurology · 2026Pooled it
- Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis.Journal of neuromuscular diseases · 2024Pooled it
- Barriers and facilitators to the implementation of guidelines in rare diseases: a systematic review.Orphanet journal of rare diseases · 2023Pooled it
- Duchenne muscular dystrophy in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact.PloS one · 2023Pooled it
- The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review.Orphanet journal of rare diseases · 2021Pooled it
- Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis.European journal of epidemiology · 2020Pooled it
- Guidance for the care of neuromuscular patients during the COVID-19 pandemic outbreak from the French Rare Health Care for Neuromuscular Diseases Network.Revue neurologique · 2020Guideline
- Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.Pharmacological research · 2018Trial
- Exploring Awareness of and Self-Reported Adherence to Neuromuscular Clinical Practice Guidelines Among Australian and New Zealand Health Professionals: A Cross-Sectional Survey Study.Journal of evaluation in clinical practice · 2026Article
- Genetic, Clinical, and Management Characteristics of Duchenne Muscular Dystrophy in Saudi Arabia.Healthcare (Basel, Switzerland) · 2026Article
- Caregiver burden in Duchenne muscular dystrophy in Europe, Japan, and the United States - a real-world study.BMC neurology · 2026Article
- Systematic Literature Reviews of Health-State Utility Values, Costs, and Healthcare Resource Use in Duchenne Muscular Dystrophy.Journal of health economics and outcomes research · 2026Article
- The Importance of Early Diagnosis and Treatment to Limit the Impact of Dystrophic Cardiomyopathy-We've Seen This Film Before, and We Didn't Like the Ending.Journal of cardiovascular development and disease · 2025Review
- Epidemiology, disease burden and costs of Duchenne muscular dystrophy in Germany: an observational, retrospective health claims data analysis.Orphanet journal of rare diseases · 2025Observational
- An underestimated factor for therapeutic decision-making in rare diseases: parents' (un)knowledge-the example of Duchenne muscular dystrophy caregivers and non-invasive ventilation.Orphanet journal of rare diseases · 2025Article
- Texture analysis of cardiovascular MRI native T1 mapping in patients with Duchenne muscular dystrophy.Orphanet journal of rare diseases · 2025Article
- Patient demographics, clinical characteristics and genetic mutations of DMD and BMD patients in Qatar Epidemiological and genetic profile of Duchenne muscular dystrophy and Becker muscular dystrophy patients in Qatar: a retrospective cohort study.Frontiers in pediatrics · 2025Article
- Title-molecular diagnostics of dystrophinopathies in Sri Lanka towards phenotype predictions: an insight from a South Asian resource limited setting.European journal of medical research · 2024Article
- Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015-2022): 2022 interim analysis.Journal of neurology · 2023Article
- Time to diagnosis of Duchenne muscular dystrophy in Austria and Germany.Scientific reports · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
25 authors at 7 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPublication of comprehensive clinical care guidelines for Duchenne muscular dystrophy (DMD) in 2010 was a milestone for DMD patient management. Our CARE-NMD survey investigates the neuromuscular, medical, and psychosocial care of DMD patients in Europe, and compares it to the guidelines.
methodsA cross-sectional survey of 1677 patients contacted via the TREAT-NMD patient registries was conducted using self-report questionnaires in seven European countries.
resultsSurvey respondents were 861 children and 201 adults. Data describe a European DMD population with mean age of 13.0 years (range 0.8-46.2) of whom 53% had lost ambulation (at 10.3 years of age, median). Corticosteroid medication raised the median age for ambulatory loss from 10.1 years in patients never medicated to 11.4 years in patients who received steroids (p < 0.0001). The majority of patients reported receiving care in line with guidelines, although we identified significant differences between countries and important shortcomings in prevention and treatment. Summarised, 35% of patients aged≥ nine years received no corticosteroid medication, 24% of all patients received no regular physiotherapy, echocardiograms were not performed regularly in 22% of patients, pulmonary function was not regularly assessed in 71% of non-ambulatory patients. Patients with regular follow-up by neuromuscular specialists were more likely to receive care according to guidelines, were better satisfied, and experienced shorter unplanned hospitalization periods.
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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.