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鈥檚 Hospital Medical Center, she trained in Pediatric Pulmonology at Boston Children鈥檚 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.
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a鈥�
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be鈥�
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