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.
Understanding the Gene Therapy Process and Aftercare is the fourth webinar in an exciting five-part series on gene therapy from NORD in collaboration with the American Society for Gene and Cell Therapy (ASGCT.) Join us for upcoming topics in the series include the following.
NORD thanks AVROBIO, Amicus, Bluebird Bio and Sarepta for their support of this educational resource.