by Madaline Spencer | May 11, 2026
Amol Sura, MD, Foster Center for Ocular Immunology, Dept of Ophthalmology, Duke University, Durham, North Carolina, discusses the diagnosis and management of plasminogen deficiency, a rare condition in which the eyes are first affected, but it manifests in mucous...
by Madaline Spencer | May 11, 2026
William W. Motley, MD, RAP-219 Program Leader at Rapport Therapeutics, discusses follow-up data of RAP-219 for the treatment of focal onset seizures. Focal onset seizures refer to abnormal neural activity in one brain area within one brain hemisphere...
by Madaline Spencer | May 8, 2026
Brian Moseley, MD, Senior Medical Director at UCB, discusses the GEMZ clinical trial of fenfluramine in patients with CDKL5 deficiency disorder (CDD). CDD is a rare genetic disorder characterized by difficult‑to‑control seizures and severe neuro‑developmental...
by Madaline Spencer | May 6, 2026
The US Food and Drug Administration (FDA) has approved Jakafi XR (ruxolitinib) extended-release tablets for the treatment of three rare hematologic conditions. The indications include the treatment of: (1) adults with intermediate- or high-risk myelofibrosis (MF); (2)...
by Madaline Spencer | May 5, 2026
Dena Sherwood, mother to a neuroblastoma survivor and Founder of Arms Wide Open Childhood Cancer Foundation, discusses CureFest 2026. Ms. Sherwood and her family founded the Arms Wide Open Childhood Cancer Foundation following the struggles their son...
