Understanding the Gene Therapy Process and Aftercare

During this webinar, clinicians who deliver potentially life-changing gene therapies will explain the gene therapy process and aftercare. Dr. Jerry Mendell, Neurologist and Principal Investigator for the Center for Gene Therapy at Nationwide Children’s Hospital and Dr. Barry Byrne, Associate Chair of Pediatrics and Director of the University of Florida Powell Center and Child Health Research Institute explain how patients are identified as candidates for gene therapy, who may be on a gene therapy treatment team, how long the procedure takes and what to expect during recovery and longer term follow up.


Webinar Speakers
Dr. Jerry Mendell has dedicated his clinical focus to neuromuscular diseases for the past five decades. For the past 15 years, he has worked to establish the Neuromuscular Gene Therapy Program at Nationwide Children’s Hospital. He recruited gifted scientists to join the effort to find treatments for fatal muscle and nerve diseases using gene therapy tools. His efforts in SMA, Duchenne and limb girdle muscular dystrophies have been particularly rewarding. Translation of research from the bench to the bedside is now having a significant impact on these diseases and affected patients.


Dr. Barry Byrne is a clinician scientist who studies a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. His focus as a pediatric cardiologist 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. They develop new therapies using Adeno-Associated Virus mediated gene therapy to restore cardiac and skeletal muscle function in DMD, Friedreich’s Ataxia, Pompe, and other inherited neuromuscular diseases.


Ms. Katie Kowalski is the Senior Program Manager for NORD’s Educational Initiatives. In that role, she works to develop and implement educational programs for medical professionals, patients and their families. She serves as a Program Manager for key programs within NORD’s Education Department including continuing medical education, the educational webinar series and student programs.

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