by Victoria Radke | Aug 30, 2021
The Food and Drug Administration (FDA) has approved MK-6482 (belzutifan) for adult patients with von Hippel-Lindau disease who require treatment for associated renal cell carcinoma, central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors...
by Victoria Radke | Aug 27, 2021
Richard Lafayette, MD, FACP, Director of the Stanford Glomerular Disease Center, Editor-in-Chief of ASN Kidney News, and Rheumatologist at Stanford Health Care, describes the current standard of care for immunoglobulin A (IgA) nephropathy. IgA nephropathy,...
by Victoria Radke | Aug 26, 2021
The Food and Drug Administration (FDA) announced the approval of ivosidenib (Tibsovo) for advanced or metastatic cholangiocarcinoma with an isocitrate dehydrogenase-1 (IDH1) mutation. Cholangiocarcinoma is a rare cancer that forms in the bile duct. Patients with...
by Victoria Radke | Aug 24, 2021
The Social Security Administration has recently announced an expansion of its Compassionate Allowances List (CAL) to include nine additional rare disease populations, including adults with Duchenne Muscular Dystrophy, Charlevoix Saguenay Spastic Ataxia (ARSACS),...
by Victoria Radke | Aug 16, 2021
The wait for a new therapy to treat fibrodysplasia ossificans progressiva (FOP) just got longer. Ipsen announced they have withdrawn their New Drug Application (NDA) with the Food and Drug Administration (FDA) for the orphan drug palovarotene to treat FOP. FOP is an...
