by Madaline Spencer | Aug 13, 2024
James “Brad” Elder, MD, Director of Neurosurgical Oncology at the Ohio State University Comprehensive Cancer Center, discusses a clinical trial evaluating the investigational gene therapy, AVB-101 in patients with frontotemporal dementia (FTD). FTD is a...
by Madaline Spencer | Aug 12, 2024
Kelly Knupp, MD, Associate Professor of Pediatrics and Neurology at the University of Colorado, discusses supplemental data on the efficacy of fenfluramine in treating Dravet syndrome-associated seizures. Dravet syndrome is the most severe of a group of...
by Scott Harwood | Aug 9, 2024
Hideki Garren, MD, PhD, Chief Medical Officer for Prothena, discusses the pathophysiology of AL amyloidosis and the mechanism of action of investigational drug birtamimab. Amyloid light chain (AL) amyloidosis is a progressive plasma cell disorder in...
by Madaline Spencer | Aug 7, 2024
Jula Inrig, MD, Chief Medical Officer at Travere Therapeutics, discusses a clinical trial that tested sparsentan for treatment of IgA nephropathy, compared to traditional RAS inhibitors. IgA nephropathy (IgAN) is a rare kidney disorder that occurs when...
by Scott Harwood | Aug 6, 2024
Alaa Hamed, Global Head of Medical Affairs for Rare Diseases for Sanofi, discusses two clinical trials that reinforce positive data for hemophilia treatments. Hemophilia is a rare bleeding disorder that slows the blood clotting process. People with this...
