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Dr. Barry Byrne is the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. After obtaining a B.S. degree in Chemistry from Denison University, he pursued his medical education, as well as a Ph.D. in Microbiology and Immunology, at the University of Illinois. He completed his pediatric residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at Johns Hopkins University. Joining the University of Florida in 1997, he has served in a variety of clinical, research and educational roles, and is now the Earl and Christy Powell University Chair in Genetics.
Dr. Byrne is currently conducting numerous clinical research studies for a variety of rare diseases, with specific attention to developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness, cardiac dysfunction and respiratory dysfunction. His research team has made significant contributions to the understanding and treatment of Pompe disease, a type of muscular dystrophy resulting from abnormal glycogen accumulation in the muscle. His current research has focused on developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe, Duchenne muscular dystrophy and other inherited myopathies.
This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2…
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
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— Dr. Barry J ByrneOur team provides fully integrated research and innovative clinical care in rare diseases where there may be no alternative therapies.
December 6, 2023
Two proposals from the University of Florida College of Medicine — one that aims to build a fully immersive digital replica of operating rooms and intensive…
Department of Pediatrics, +6 more
November 22, 2023
Gene therapy could become safer and more effective by calming the inflammation that occurs when the treatment is administered, University of Florida Health…
Department of Pediatrics, +3 more
