by Madaline Spencer | Apr 23, 2026
Fernanda Leal-Pardinas, MD, MSc, Global Clinical Development Lead, Rare & Metabolic Disorders at Otsuka Pharmaceutical, discusses the open-label extension data from their study testing repinatrabit for patients with phenylketonuria (PKU). PKU is a...
by Madaline Spencer | Apr 22, 2026
A retrospective observational study published in the International Journal of Cardiology evaluated a new clinical staging of hypertrophic cardiomyopathy in Fabry disease. Fabry disease is a lysosomal disorder caused by mutations in the GLA gene. It is characterized by...
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...
