by Madaline Spencer | Mar 27, 2024
Priya Kishnani, MD, Professor of Pediatrics at Duke University Medical Center, discusses intrauterine treatment for lysosomal diseases. The study, “Measurement of glycosaminoglycans in the amniotic fluid of fetuses with mucopolysaccharidoses treated in a...
by James Radke, PhD | Mar 26, 2024
Dean Suhr, President and Co-Founder of MLD Foundation, discusses current and emerging treatment options in metachromatic leukodystrophy (MLD). MLD is a genetic lysosomal disorder characterized by the toxic buildup of lipids and other storage materials...
by Madaline Spencer | Mar 25, 2024
F. Sessions Cole, Secretary, UDNF and Professor of Pediatrics, Washington University School of Medicine, and Michele Herndon, MSN, RN and Program Director of UDNF’s Patient Navigation Program, discuss the Undiagnosed Diseases Network Foundation and their Patient...
by Madaline Spencer | Mar 21, 2024
Trevor Baglin, MD, PhD, Vice President of Centessa Pharmaceuticals UK, discusses SerpinPC, an investigational drug candidate, for the potential treatment of severe hemophilia. Hemophilia is a bleeding disorder that slows the blood clotting process....
by Madaline Spencer | Mar 21, 2024
Matt Trudeau, head of ITF Therapeutics, discusses givinostat, an investigational therapy for Duchenne muscular dystrophy (DMD). DMD is a rare neuromuscular disease caused by genetic variants in the DMD gene. The disease primarily affects the muscles,...
