by James Radke, PhD | Apr 5, 2023
Pramod Mistry, MD, PhD, Professor of Medicine at Yale University School of Medicine, provides an overview on how persons with Gaucher disease type 3 are managed. Gaucher disease is a rare lysosomal disorder due to reduced levels of glucocerebrosidase that...
by James Radke, PhD | Apr 3, 2023
Raphael Schiffmann, MD, of 4D Molecular Therapeutics, discusses the cardiac effects of 4D-310 in adults with Fabry disease. Fabry disease is a rare lysosomal disorder that results in the buildup of globotriaosylceramide. Characteristic features of Fabry...
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...
