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Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the…
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with…
19 publications
2024
Pediatric Pulmonology
Publisher's site2024
Pediatric Pulmonology
Publisher's site2024
The Journal of clinical investigation
PubMed • Publisher's site2024
Advances in Medical Education and Practice
Publisher's site2023
Pediatric Pulmonology
Publisher's site