by Madaline Spencer | Dec 9, 2025
Kim Moran, Head of U.S. Rare Diseases at UCB, discusses the approval of Kygevvi (doxecitine and doxribtimine) for patients with thymidine kinase 2 deficiency (TK2d). TK2d is a rare genetic disease characterized by progressive and severe muscle weakness....
by Madaline Spencer | Dec 8, 2025
Michelle Rheault, MD, Pediatric Nephrologist at the University of Minnesota, discusses data from the DUPLEX study testing sparsentan to treat patients with focal segmental glomerulosclerosis (FSGS). FSGS is a rare kidney disorder characterized by...
by Madaline Spencer | Dec 6, 2025
The U.S. Food and Drug Administration (FDA) has approved Voyxact (sibeprenlimab) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. IgAN is a kidney disorder that occurs when IgA protein...
by Madaline Spencer | Dec 5, 2025
The U.S. Food and Drug Administration (FDA) has approved Itvisma (onasemnogene abeparvovec) for the treatment of patients ages two years and older with spinal muscular atrophy (SMA) with a confirmed mutation in the SMN1 gene. The gene therapy is administered...
by Madaline Spencer | Dec 5, 2025
Ghaith Noaiseh, MD, Associate Professor of Allergy, Clinical Immunology, and Rheumatology at The University of Kansas Medical Center, discusses the development of nipocalimab for the treatment of Sjögren’s disease (SjD). SjD is an autoimmune disorder in...
