Neurology. Genetics · 2026
Duchenne Muscular Dystrophy and Delandistrogene Moxeparvovec Gene Therapy in Children: A Systematic Review and Meta-Analysis.
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Antonello Breno Bopp, Cargnelutti Fontoura Fabio, Braga Albuquerque Anna Luiza, Giovacchini Giovanna, Grespan Dill Laura, Valadão Maria Clara, Hadj Sadok Sara, Zattar Ribeiro Paulo Victor
Аннотация
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, p = 0.04) and TTR (MD = -0.85, p < 0.01). Sensitivity analysis demonstrated improved 10MWR (MD = -0.71, p = 0.02). Muscle dystrophin content significantly increased (MD = 28.39, p < 0.01).
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.
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, p = 0.04) and TTR (MD = -0.85, p < 0.01). Sensitivity analysis demonstrated improved 10MWR (MD = -0.71, p = 0.02). Muscle dystrophin content significantly increased (MD = 28.39, p < 0.01).
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.