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Pediatric Neuromuscular Rehabilitation Specialist, Pediatric Pulmonologist (Child Lung Specialist)
Carmen Leon Astudillo, MD, is a board-certified pediatric pulmonologist. She received her medical degree from Universidad del Azuay, in Ecuador. After completing her pediatric residency at Cincinnati Children’s Hospital Medical Center, she trained in Pediatric Pulmonology at Boston Children’s Hospital. Dr. Leon Astudillo joined the University of Florida as a clinical assistant professor in 2018. Her clinical and research duties focus mainly on patients with neuromuscular and rare diseases, in particular those with spinal muscular atrophy and Duchenne muscular dystrophy. Dr Leon Astudillo is also part of the aerodigestive program at the University of Florida.
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…
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