by Madaline Spencer | Apr 9, 2025
The U.S. Food and Drug Administration (FDA) has approved Uplizna (inebilizumab-cdon) for the treatment of adults with immunoglobulin G4-related disease (IgG4-RD). IgG4-RD IgG4-RD is an immune-mediated condition that can affect multiple organ systems. Common...
by Madaline Spencer | Apr 9, 2025
This study evaluates the characterization of this atypical phenotype and focuses on 75 patients with ADPKD-like phenotypes with 41 different monoallelic predicted loss-of-function IFT140 variants. Autosomal dominant polycystic kidney disease (ADPKD) is caused by gene...
by Madaline Spencer | Apr 8, 2025
The U.S. Food and Drug Administration (FDA) has granted accelerated approval of Vanrafia (atrasentan) for the treatment of adults with primary immunoglobulin A nephropathy (IgAN). IgA Nephropathy IgAN is a rare disorder that occurs when IgA accumulates in the kidneys....
by Madaline Spencer | Apr 8, 2025
Ashley Shoemaker, MD, Associate Professor of Pediatrics and Pediatric Endocrinology at Vanderbilt University, discusses the approval of Vykat XR (diazoxide choline) for the treatment of hyperphagia in patients ages 4 years and older with Prader-Willi syndrome (PWS)....
by Madaline Spencer | Apr 7, 2025
A recent study identified three clinical subgroups of patients with anti-SRP immune mediated necrotizing myopathy (anti-SRP IMNM). Anti-SRP IMNM is a rare autoimmune disorder characterized by muscle weakness and necrosis in the proximal muscles. The exact cause of...
