by Madaline Spencer | Apr 24, 2026
Richard J. Nowak, MD, global principal MINT investigator and director of the Myasthenia Gravis Clinic at Yale University, discusses a post-hoc analysis of Uplizna (inebilizumab) on the effect of ocular manifestations in generalized myasthenia gravis (gMG). gMG...
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...
