by Madaline Spencer | Apr 21, 2026
Ben Zimmer, CEO of Priovant Therapeutics, discusses results from the recent phase 3 study testing brepocitinib for the treatment of dermatomyositis (DM). DM is a rare autoimmune condition that causes skin changes and muscle weakness. Symptoms can include...
by Madaline Spencer | Apr 17, 2026
Dena Sherwood, mother to a neuroblastoma survivor and Founder of Arms Wide Open Childhood Cancer Foundation, discusses her organization and the CureFest event. Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor...
by Madaline Spencer | Apr 16, 2026
The US Food and Drug Administration (FDA) has approved Filspari (sparsentan) to reduce proteinuria in patients with focal segmental glomerulosclerosis (FSGS). The indication includes adult and pediatric patients ages 8 years and older without nephrotic syndrome. In...
by Madaline Spencer | Apr 15, 2026
Jennifer Ibrahim, Head of North America, Rare Disease Medical Affairs at Sanofi, discusses the Breakthrough Therapy designation of venglustat for the treatment of neurological manifestations in Gaucher disease type 3 (GD3). Gaucher disease refers to a...
by Madaline Spencer | Apr 14, 2026
Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development. On November 12, 2025, the US Food and Drug Administration (FDA) released the...
