by Madaline Spencer | Aug 7, 2026
The US Food and Drug Administration (FDA) has approved Orzeyful (oveporexton) for the treatment of adults with narcolepsy type 1 Narcolepsy is a chronic neurological disorder characterized by an inability of the brain to control sleep-wake cycles. Patients with...
by Madaline Spencer | Aug 6, 2026
Ryan Bucco, PharmD, Vice President of Medical Affairs in Rare Disease at Acadia, discusses the LOTUS study in patients with Rett syndrome treated with Daybue (trofinetide). Rett syndrome is a neurodevelopmental condition that primarily affects girls. People...
by Madaline Spencer | Aug 5, 2026
Bruno Fattizzo, MD, Department of Oncology and Hemato-Oncology at the University of Milan, discusses results from the ENERGY clinical trial of Imaavy (nipocalimab) in warm autoimmune hemolytic anemia (wAIHA). wAIHA is a rare hematologic autoimmune condition...
by Madaline Spencer | Aug 4, 2026
Doug Kerr, MD, PhD, MBA, Chief Medical Officer of Dyne Therapeutics, discusses the initiation of the phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51...
by Madaline Spencer | Jul 31, 2026
Pete Schmidt, MD, MSc, Chief Medical Officer of Portal Therapeutics, discusses data from the phase 2a GATEWAY trial testingPORT-77, an ABCG2 inhibitor, in patients with erythropoietic protoporphyria (EPP). EPP is a rare congenital metabolic disorder...
