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...
by James Radke, PhD | Mar 15, 2023
Jonathan Barratt, PhD, FRCP, Professor of Renal Medicine, University of Leicester, UK, gives an overview of immunoglobulin A (IgA) nephropathy. As Dr. Barratt explains, IgA nephropathy is a rare kidney disease characterized by build-up of IgA deposits in the...
by James Radke, PhD | Mar 2, 2023
Allen Davidoff, PhD, CEO of XORTX Therapeutics, discusses two phase 3 studies set to launch in the next two years evaluating oxypurinol in patients with autosomal dominant polycystic kidney disease (ADPKD). ADPKD is a genetic disorder characterized by the...
by James Radke, PhD | Feb 24, 2023
The U.S. Food and Drug Administration has approved efanesoctocog alfa for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. Angela Weyand,...
