SynthesisJournal of neuromuscular diseases2026
The natural history of Becker muscular dystrophy: A systematic literature review.
Synthesis in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Not yet cited in PubMed.
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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.
Functional Muscle Ischemia and PDE5A Inhibition in Becker Muscular Dystrophy
Does Modulation of the nNOS System in Patients With Muscular Dystrophy and Defect nNOS Signalling Affect Cardiac, Muscular or Cognitive Function?
Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
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6 authors.
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Abstract
backgroundBecker muscular dystrophy (BMD) is caused primarily by in-frame mutations in the
methodsA systematic literature review (SLR) was refreshed in 2022 using MEDLINE and EMBASE to identify articles describing the natural history of BMD. The proportion of patients experiencing clinical milestones was reported by 'life-stage' age groups (0-17; 18-40; 41+ years) using patient-level data from the general BMD population; age at each milestone's occurrence as mean (standard deviation [SD]).
resultsFrom 4948 abstracts screened, 121 publications were included. Among 36 general BMD population studies, by age 41+ years (lifetime-risk proxy), 93.6% experienced muscle weakness; 69.4% cardiac involvement; 55.6% scoliosis; 47.4% loss of ambulation; and 33.3% ventilation. Decreased cognitive function or cognitive dysfunction were reported in 41% across all ages. Among those experiencing milestones (79 studies), mean (SD) age at symptom onset was 12.5 (9.7); muscle weakness, 19.9 (11.7); scoliosis, 24.9 (3.1); cardiac involvement, 31.9 (13.4); loss of ambulation, 33.3 (13.5); ventilation, 35.7 (12.2); and death at 55.6 (19.4) years. Data availability ranged from three to 1079 patients/outcome.
conclusionsThis SLR highlights the variability in disease presentation in BMD. Stratifying BMD populations into phenotype groups based on the full spectrum of clinical manifestations may better capture disease progression and enhance comparability across studies.Included clinical trials:ClinicalTrials.gov NCT01070511 (https://clinicaltrials.gov/study/NCT01070511), ClinicalTrials.gov NCT02147639 (https://clinicaltrials.gov/study/NCT02147639?term=Becker%20Muscular%20Dystrophy&intr=Sodium%20Nitrate&rank=3), ClinicalTrials.gov NCT01350154 and EudraCT number: 2010-024659-10 (https://clinicaltrials.gov/study/NCT01350154?term=NCT01350154&rank=1; https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-024659-10/results).
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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 Socratic graph.