How Homocystinuria Is Currently Treated

  Can Ficicioglu, MD, PhD, Clinical Director of the Metabolic Disease Program at Children’s Hospital of Philadelphia (CHOP), discusses the management of homocystinuria. Homocystinuria is a rare congenital metabolic disease characterized by extremely...

Zero Relapses in Patients With NMOSD Given Ravulizumab

  Sean J. Pittock, MD, Director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo’s Neuroimmunology Laboratory discusses the latest results from Phase III CHAMPION-NMOSD trial recently presented at European Committee...

Dravet Syndrome Clinical Trial With STK-001 Continues

  Barry S. Ticho, MD, PhD, Chief Medical Officer at Stoke Therapeutics, discusses the phase 1/2a MONARCH study, which is evaluating STK-001 in patients with Dravet syndrome. As Dr. Ticho explains, Dravet syndrome is a rare neurological condition that usually...