by James Radke, PhD | Mar 29, 2023
Paul Orchard, MD, from the University of Minnesota Medical School, discusses the compassionate use of gene therapy in children with metachromatic leukodystrophy (MLD). MLD is a lysosomal disorder characterized by the accumulation of sulfatides in cells....
by James Radke, PhD | Mar 27, 2023
Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the two annual events Duke University hosts for patients with Pompe disease. Pompe disease is a rare inherited lysosomal disorder due to inadequate levels of acid...
by James Radke, PhD | Mar 24, 2023
Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses ongoing study looking to assess the natural history of babies diagnosed with later-onset Pompe disease. Pompe disease is a rare inherited lysosomal disorder due to an...
by James Radke, PhD | Mar 20, 2023
Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the latest long-term results from the COMET study involving late-onset Pompe disease. Pompe disease is a lysosomal disorder caused by inadequate levels of...
by James Radke, PhD | Mar 16, 2023
Priya Kishnani, MD, Professor of Pediatrics at the Duke University School of Medicine, discusses the difference types of Pompe disease. Pompe disease is a rare inherited lysosomal disorder due to inadequate levels of acid alpha-glucosidase (GAA) that results...
