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 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 22, 2024
The U.S. Food and Drug Administration (FDA) has approved givinostat for the treatment of patients with all genetic variants of Duchenne muscular dystrophy (DMD). DMD is caused by genetic changes in the DMD gene that leads to a reduction in functioning levels of the...
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,...
