by Madaline Spencer | Feb 19, 2025
Dina Matos, Executive Director of the CARES Foundation, discusses the recent approval of crinecerfont to treat patients with congenital adrenal hyperplasia (CAH) by the U.S. Food and Drug Administration (FDA). CAH refers to a group of genetic conditions...
by Madaline Spencer | Feb 18, 2025
The U.S. Food and Drug Administration (FDA) has approved vimseltinib for adult patients with symptomatic tenosynovial giant cell tumor (TGCT). TGCT is a rare disease in which the tissue lining the joints and tendons in the body grows abnormally. It is characterized by...
by Madaline Spencer | Feb 18, 2025
Michael Panzara, MD, Chief Medical Officer at Neurvati Neurosciences and GRIN Therapeutics, discusses topline results of the Honeycomb trial for GRIN-related neurodevelopmental disorder with gain-of-function (GoF) variants. GRIN-related neurodevelopmental...
by Madaline Spencer | Feb 17, 2025
Hugues Chanteaux, PhD, Quantitative Clinical Pharmacology Lead at UCB, discusses results from a study evaluating two different routes of alprazolam administration, inhaled (“Staccato”) versus oral administration in patients with acute seizures. ...
by Madaline Spencer | Feb 16, 2025
Gus Baker, PhD, International Bureau for Epilepsy, discusses a social media listening study looking at the experiences of women of childbearing age with epilepsy. A social media listening study was conducted to identify key topics of conversation, unmet...
