• Understanding Neuromuscular Diseases: 

    What You Need To Know

Dr. Barry J Byrne

Chief Medical Advisor, Muscular Dystrophy Association



Dr. Barry J Byrne can address:

(1) The impact of newborn screening on babies born with a neuromuscular disease; (2) How MDA’s strategic funding has impacted SMA research and treatments; (3) How MDA offers care for people living with SMA; (4) MDA’s advocacy efforts to ensure people with SMA have better access to care and therapies; (5) treatment options for Pompe and how MDA has contributed to those research efforts; (6) MDA Care Center Network.

Dr. Barry J Byrne, Chief Medical Advisor, Muscular Dystrophy Association Bio:

Dr. Barry Byrne is the Chief Medical Advisor at the Muscular Dystrophy Association as well as 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 cardiac 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 muscle and motor units. The research team has been developing new therapies using AAV-mediated gene therapy to restore muscle function in Pompe, DMD, Friedreich’s ataxia and other inherited neuromuscular diseases.

The group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing. These strategies are also being applied in Duchenne Muscular Dystrophy (DMD) and Friedreich’s Ataxia (FA), which are in the late stages of clinical development. The work is supported by several NIH and foundation awards. Dr. Byrne is the Associate Chair of Pediatrics and Director of the University of Florida Powell Center. He obtained his bachelor’s degree from Denison University, his medical and doctorate degrees from the University of Illinois, and completed his pediatrics residency and cardiology fellowship as well as post-doctoral training in biological chemistry at the Johns Hopkins Hospital. He joined the University of Florida in 1997 and is now the Early and Christy Powell University Chair in Genetics. 

Dr. Barry J Byrne is an ardent supporter of newborn screening and has been an innovator and early adopter of new FDA-approved therapies that have the potential to alter the course of some neuromuscular disorders. He was the first physician to administer an approved gene therapy to a neuromuscular patient in the U.S.

Quotes from Dr. Barry J Byrne

“We are just beginning to realize the impact of the current revolution in the treatment of neuromuscular disorders. Newborn screening and access to newly-approved therapies are changing patients’ lives when diagnosed with neuromuscular diseases. Disease-modifying therapies which are available today are meeting current needs and not just imagined in the future. There is an urgent need to bring innovative care, cutting-edge clinical research and new breakthrough treatments to the community.” – Muscular Dystrophy Association 

“The opportunity for gene therapy to transform the life of a patient with a genetically defined disease is unique. In the history of medicine, there have been very few treatments of any disease that can effectively be called curative. In some circumstances, this will be possible with gene therapy. It’s rewarding to be able to provide such a treatment to patients.” – MFRF Foundation

Other Muscular Dystrophy Association Spokespeople Include:

  • Donald S. Wood, PhD, President and CEO, Muscular Dystrophy Association
  • Kristine Welker, Chief of Staff, Muscular Dystrophy Association
  • Dr. Sharon Hesterlee, Executive Vice President, Chief Research Officer, Muscular Dystrophy Association
  • Paul Melmeyer, Vice President, Public Policy and Advocacy, Muscular Dystrophy Association
  • Mindy Henderson, Director, Quest Editor-in-Chief, Muscular Dystrophy Association


  • Newborn screening
  • Spinal muscular atrophy (SMA)
  • MDA’s advocacy efforts
  • MDA Care Center Network
  • Pompe disease

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