Barry Byrne, MD, PhD

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.

Exon Skipping and Read Through Agents Panel

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.

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

Jay Chauhan, PharmD

Medical Science Liaison

NS Pharma

Brett Crawford, PhD

Executive Director and Head of the Musculoskeletal Therapeutic Area

BioMarin Pharmaceutical Inc.

Ash Dugar, PhD

Senior Vice President, Medical Affairs

Dyne Therapeutics

Xiao Shelley Hu, PhD, MS

VP

Wave Life Sciences

Matthew Klein, M.D., M.S., FACS

Chief Operating Officer

PTC Therapeutics

Nerissa Kreher, MD, MBA

Chief Medical Officer

Entrada Therapeutics

James McArthur, PhD

President and Chief Executive Officer

PepGen

Mark Stahl, MD, PhD

Senior Medical Director, Clinical Development

Avidity Biosciences

Deanna Tucker, PharmD

Senior Medical Science Liaison

Sarepta Therapeutics

Pillars of the Early Journey

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.

Barry Byrne, MD, PhD

Professor and Associate Chair

University of Florida, Department of Pediatrics

Diana Castro, MD

Associate Professor of Pediatrics, Neurology and Neurotherapeutics

University of Texas Southwestern

Kindann Fawcett, PhD

Post Doctoral Fellow

University of Arkansas for Medical Sciences