by Madaline Spencer and Ailene Cantelmi | Oct 16, 2026
Jane Salmon, MD, Professor of Medicine at Weill Cornell Medical College, discusses antiphospholipid syndrome (APS), its impact on pregnancy, and emerging research into Cimzia (certolizumab pegol) as a potential approach to preventing serious pregnancy complications. ...
by CheckRare Staff and Ailene Cantelmi | Oct 14, 2026
In this episode, we discuss Ultragenyx’s Priority Review Voucher that sold for $210 million, the efficacy of newborn screening on MLD, and ethical considerations needed to design and conduct clinical trials. Ultragenyx sells PRV for $210M This past week,...
by Madaline Spencer and Ailene Cantelmi | Oct 14, 2026
Keisha L. Gibson, MD, MPH, pediatric nephrologist at the UNC School of Medicine, discusses the approval of Gazyva (obinutuzumab) for patients with childhood-onset idiopathic nephrotic syndrome (INS). INS is a rare, autoimmune kidney disease that is often...
by CheckRare Staff and Ailene Cantelmi | Oct 12, 2026
Jorge Nieva, MD, Associate Professor at the Keck School of Medicine, discusses new analyses from the TRUST studies evaluating taletrectinib in patients with ROS1-positive non-small cell lung cancer (NSCLC). ROS1-positive NSCLC is a rare molecular subtype of...
by CheckRare Staff and Ailene Cantelmi | Oct 8, 2026
On September 28th, the FDA approved Emcitate, or tiratricol, from Egetis Therapeutics. It’s the first-ever treatment for MCT8 deficiency, a condition also known as Allan-Herndon-Dudley syndrome. MCT8 deficiency is a rare genetic disorder that mostly affects...
