ArticleJournal of neuromuscular diseases2024
Findings from the Longitudinal CINRG Becker Natural History Study.
Article in Journal of neuromuscular diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
What it found
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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.
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Who cites it
7 citing papers in PubMed.
- Total body and appendicular lean mass by dual-energy X-ray absorptiometry are highly associated with muscle function and bone mineral density in Becker muscular dystrophy.Journal of neuromuscular diseases · 2026Article
- Sensitivity of Different Clinical Outcome Measures in Assessing Adults With Becker Muscular Dystrophy: A 3-Year Natural History Study.Neurology · 2025Article
- Longitudinal Changes of Motor Function in Becker Muscular Dystrophy.Neurology. Genetics · 2025Article
- Characterisation of a large, single-centre cohort of patients with Becker muscular dystrophy to inform standardised care guidelines.Journal of neurology · 2025Article
- The Persistence of Duchenne vs Becker Muscular Dystrophies:Neurology. Genetics · 2025Article
- Multi-parameter quantitative magnetic resonance imaging for early detecting skeletal muscle involvement and predicting functional decline in children with Becker muscular dystrophy.Pediatric radiology · 2025Article
- The First Decade of Journal of Neuromuscular Diseases: Supporting and Advancing the Rapidly Evolving Field of Translational Research.Journal of neuromuscular diseases · 2024Article
Corrections and comments
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Authors and funding
20 authors.
Funding
Abstract
backgroundBecker muscular dystrophy is an X-linked, genetic disorder causing progressive degeneration of skeletal and cardiac muscle, with a widely variable phenotype.
objectiveA 3-year, longitudinal, prospective dataset contributed by patients with confirmed Becker muscular dystrophy was analyzed to characterize the natural history of this disorder. A better understanding of the natural history is crucial to rigorous therapeutic trials.
methodsA cohort of 83 patients with Becker muscular dystrophy (5-75 years at baseline) were followed for up to 3 years with annual assessments. Muscle and pulmonary function outcomes were analyzed herein. Age-stratified statistical analysis and modeling were conducted to analyze cross-sectional data, time-to-event data, and longitudinal data to characterize these clinical outcomes.
resultsDeletion mutations of dystrophin exons 45-47 or 45-48 were most common. Subgroup analysis showed greater pairwise association between motor outcomes at baseline than association between these outcomes and age. Stronger correlations between outcomes for adults than for those under 18 years were also observed. Using cross-sectional binning analysis, a ceiling effect was seen for North Star Ambulatory Assessment but not for other functional outcomes. Longitudinal analysis showed a decline in percentage predicted forced vital capacity over the life span. There was relative stability or improved median function for motor functional outcomes through childhood and adolescence and decreasing function with age thereafter.
conclusionsThere is variable progression of outcomes resulting in significant heterogeneity of the clinical phenotype of Becker muscular dystrophy. Disease progression is largely manifest in adulthood. There are implications for clinical trial design revealed by this longitudinal analysis of a Becker natural history dataset.
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