Barry J. Byrne

Dr. Barry Byrne is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units.

The research team has been developing new therapies using adeno-associated virus (AAV)-mediated gene therapy to restore cardiac and skeletal muscle function in Duchenne muscular dystrophy, Friedreich’s ataxia, Pompe, and other inherited neuromuscular diseases. His group at University of Florida Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing. The work is supported by several National Institute of Health (NIH) and foundation awards. Dr. Byrne is the Associate Chair of Pediatrics, Earl and Christy Powell University Chair in Genetics and Director of the University of Florida Powell Center and Child Health Research Institute. In addition to his academic appointments, he serves as the Chief Medical Advisor of the Muscular Dystrophy Association and a member of the TREAT-NMD Advisory Committee for Therapeutics (TACT).

He obtained his BS from Denison University; his MD and PhD from the University of Illinois; and completed his pediatrics residency, cardiology fellowship and post-doctoral training in biological chemistry at the Johns Hopkins Hospital.