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 Madaline Spencer | Aug 8, 2024
Pisit “Duke” Pitukcheewanont, MD, Chief Medical Officer for Lumos Pharma, discusses a phase 2 trial evaluating LUM-201 (ibutamoren) for treatment of growth hormone deficiency. Pediatric growth hormone deficiency (PGHD) is a rare condition characterized...
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 Madaline Spencer | Aug 6, 2024
The U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee voted in favor of the data supporting arimoclomol as an effective treatment for patients with Niemann-Pick disease type C (NPC). NPC is a rare lysosomal storage disease...
