by CheckRare Staff | Oct 21, 2026
On this episode, we discuss the FDA approval of Enspryng for TED, the new FDA Rare Disease Framework Initiative, Ultrangenyx’s second sold PRV this month, and using AI to reduce diagnostic delays. FDA Approves Treatment for TED The FDA has approved...
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 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...
by CheckRare Staff | Oct 1, 2026
October is Rett Syndrome Awareness Month. Rett syndrome is a neurodevelopmental condition that primarily affects girls. People with the disease appear to have normal psychomotor development during the first 6 to 18 months of life before developing severe problems with...
