Gene therapy for Pompe disease

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

 

Dwight’s faculty page

ClinicalTrials.gov listing for Pompe gene therapy trial

Dr. Kishnani’s faculty page

Actus Therapeutics

“Gene therapy trial for Pompe disease begins” DCRI press release, February 5, 2019

Click to Download: Radio In Vivo April 24, 2019