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NORD Webinar – Understanding the Gene Therapy Process and Aftercare

November 20, 2019 @ 2:00 pm - 3:00 pm

During this webinar, clinicians who deliver potentially life-changing gene therapies will explain the gene therapy process and aftercare. Don’t miss this exciting opportunity to hear 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. They will 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.
This webinar is perfect for patients, caregivers, advocates, students and the public.

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.

Gene Therapy Webinar Series

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.

  • Gene Therapy: Yesterday, Today and Tomorrow – Watch the archived video here.
  • The Science Behind Gene Therapy – Watch the archived video here.
  • The FDA’s Role in Gene Therapy – Watch the archived video here.
  • Life After Gene Therapy – Wednesday, December 18

NORD thanks AVROBIO, Amicus, Bluebird Bio and Sarepta for their support of this educational resource. 


November 20, 2019
2:00 pm - 3:00 pm
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