Guest: Dr. Dwight Koeberl, Professor of Pediatrics, Professor of Molecular Genetics and Microbiology; Medical Director, Pediatric Biochemical Genetics Laboratory, Duke University
Topics: Gene therapy clinical trial for Pompe disease, gene therapy history and current practice, enzyme replacement therapy, newborn screening, Actus Therapeutics
Dr. Dwight Koeberl
ClinicalTrials.gov listing for Pompe gene therapy trial
“Gene therapy trial for Pompe disease begins” DCRI press release, February 5, 2019
Radio In Vivo 