GRIDS Symposium Celebrates its 10th Anniversary

Ozlem Goker-Alpan, MD, Founder and Chief Medical Officer of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), discusses her work in lysosomal storage diseases and the GRIDS conference.     Lysosomal diseases are a group of rare...

New Data on Investigational Dravet Syndrome Treatment

Joseph E. Sullivan, MD, Professor of Neurology & Pediatrics at University of California San Francisco and Director of Benioff Children’s Hospital Pediatric Epilepsy Center of Excellence, discusses zovrevunersen for the treatment of patients with Dravet syndrome....

PFIC and Alagille Syndrome Studies

James Squires, MD, University of Pittsburgh, discusses recent data regarding Bylvay (odevixibat) in patients with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome.   PFIC is a heterogeneous group of autosomal recessive disorders of...