by Madaline Spencer | Aug 14, 2026
Alexandra Miller, MD, PhD, Chief of Neuro-Oncology and Co-Director of the Brain and Spine Tumor Center at the Perlmutter Cancer Center at NYU Langone Health, discusses follow-up data on the safety and efficacy of safusidenib in patients with IDH-mutant gliomas....
by Madaline Spencer | Aug 12, 2026
Guy Young, MD, Director of the Hemostasis and Thrombosis Program at Children’s Hospital Los Angeles, discusses an ATLAS-OLE subgroup analysis of fitusiran in patients with hemophilia. Hemophilia is a rare bleeding disorder that slows the blood...
by Madaline Spencer | Aug 10, 2026
Priya Kishnani, MD, Professor and Chief of the Division of Medical Genetics at Duke University, discusses the GALILEO-3 clinical trial of FLT201 in adults with Gaucher disease type 1 (GD1). GD1 is the most common form of Gaucher disease. It is caused by...
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...
