Title: Professor and Associate Chair
Organization: University of Florida, Department of Pediatrics
Bio: 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 AAV-mediated gene therapy to restore cardiac and skeletal muscle function in DMD, Friedreich’s ataxia, Pompe, and other inherited neuromuscular diseases. His group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing. Dr. Byrne is the Associate Chair of Pediatrics and Director of the University of Florida Powell Center and Child Health Research Institute. He obtained his B.S. degree from Denison University; M.D. and Ph.D. from the University of Illinois; and he completed his Pediatrics residency and cardiology fellowship as well as post-doctoral training in Biological Chemistry at the Johns Hopkins Hospital. Following his early career at Hopkins, he joined the University of Florida and is now the Earl and Christy Powell University Chair in Genetics. In addition to his academic appointments, he serves as the Chief Medical Advisor of the Muscular Dystrophy Association and a member of the TACT committee of TREAT-NMD. He is an advisor to the Pfizer Rare Disease Therapeutic Area Advisory Committee and co-founder of AavantiBio.
An analysis of combined clinical trial data and what it can teach about clinical trial design. What has gone right and what should be changed in the future?
Professor and Associate Chair
University of Florida, Department of Pediatrics
Leading Duchenne providers discuss recommendations and advancements in their specialty and answer questions from the audience.
Pediatric Neurologist
Arkansas Children's Hospital
Pediatric Gastroenterologist
Nationwide Children's Hospital
Professor and Associate Chair
University of Florida, Department of Pediatrics
A moderated panel discussion focusing on the potential challenges and complications of AAV therapy and the clinical strategies to make dosing AAV more successful.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH
Director, Center for Gene Therapy Nationwide Children's Hospital
Co-Founder and Chief Scientific Officer
Kate Therapeutics
A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Executive Director and Head of the Musculoskeletal Therapeutic Area
BioMarin Pharmaceutical Inc.
Senior Medical Director, Clinical Development
Avidity Biosciences
Senior Medical Science Liaison
Sarepta Therapeutics
Esta sesión está dirigida a familias/cuidadores de niños y adolescentes con Duchenne. Cubre cuatro pilares del viaje temprano de Duchenne: atención multidisciplinaria, esteroides, nutrición y participación en ensayos clínicos, y es seguido por una sesión de discusión/preguntas y respuestas.
Post Doctoral Fellow
University of Arkansas for Medical Sciences
Professor and Associate Chair
University of Florida, Department of Pediatrics
Pediatric Neurologist
Arkansas Children's Hospital
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern
This session is intended for families/caregivers of children and adolescents with Duchenne. It covers four pillars of the early Duchenne journey: Multidisciplinary Care, Steroids, Nutrition, and Involvement in Clinical Trials, and is followed by a discussion/Q&A session.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern
Post Doctoral Fellow
University of Arkansas for Medical Sciences