by Madaline Spencer | Feb 25, 2025
The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. CTX is a rare lipid storage disease characterized by the inability to break down certain lipids effectively. As a result,...
by Madaline Spencer | Feb 25, 2025
David Meeker, MD, Chief Executive Officer of Rhythm Pharmaceuticals, discusses the U.S. Food and Drug Administration’s (FDA) approval of setmelamotide for children 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically...
by Madaline Spencer | Feb 24, 2025
Alix Arnaud, Director of Health Economic and Value Assessment BP at Sanofi, discusses the cost of efanesoctocog alfa compared with other Factor VIII replacement therapies for major surgeries in patients with hemophilia A. Hemophilia A is an inherited...
by Madaline Spencer | Feb 21, 2025
Kelly Knupp, MD, Pediatric Epileptologist at the University of Colorado, discusses the phase 3 EMPEROR clinical trial for pediatric patients with Dravet syndrome. Dravet syndrome is the most severe of a group of conditions known as SCN1A-related seizure...
by Madaline Spencer | Feb 20, 2025
Jorge Cortes, MD, Director of the Georgia Cancer Center, discusses five year follow-up results of the OPTIC clinical trial for chronic myeloid leukemia (CML). CML is a myeloproliferative neoplasm characterized by the expression of the BCR-ABL1 fusion...
