Update your location to show providers, locations, and services closest to you.
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
2023
Current opinion in neurology
PubMed • Publisher's site2023
Frontiers in rehabilitation sciences
PubMed • Publisher's site2023
Sleep Medicine
Publisher's site2023
Molecular pharmaceutics
PubMed • Publisher's site2022
Frontiers in neurology
PubMed • Publisher's site