ReviewNeurology. Genetics2026
Duchenne Muscular Dystrophy and Delandistrogene Moxeparvovec Gene Therapy in Children: A Systematic Review and Meta-Analysis.
Review in Neurology. Genetics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the 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
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background and Objectives: Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder caused by DMD pathogenic variants, leading to dystrophin deficiency, muscle degeneration, loss of ambulation, respiratory failure, and reduced life expectancy. Current treatments, such as corticosteroids and supportive care, offer limited long-term benefits. Delandistrogene moxeparvovec is a promising gene therapy developed to restore dystrophin expression and deliver microdystrophin to skeletal and cardiac muscles. This systematic review and meta-analysis evaluate the efficacy of this treatment in ambulatory pediatric patients with DMD. Methods: A systematic search of Cochrane, PubMed, and Embase identified randomized controlled trials (RCTs) and cohort studies on delandistrogene moxeparvovec in ambulatory male children (≥4 to <8 years) with DMD. Primary outcomes included NSAA score changes, a 10-meter walk/run test (10MWR), time to rise (TTR), and dystrophin expression. Study selection followed PRISMA guidelines, and statistical analyses were conducted using R software. The study was registered in PROSPERO (CRD42025635605). Results: We included 302 participants from 4 studies (2 RCTs). Follow-up ranged from 48 weeks to 5 years, with results analyzed at 1 year. Delandistrogene moxeparvovec was administered to 107 affected individuals, while 195 were in the control group. At 1 year, the therapy significantly improved NSAA scores (MD = 2.48, Discussion: Delandistrogene moxeparvovec, despite high heterogeneity for the analysis, improved functional outcomes in ambulatory pediatric patients with DMD. Further long-term RCTs are needed to confirm its safety and efficacy.
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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.