North American Society For Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) Course: Harnessing Research and Clinical Breakthroughs in the Care of Children With Liver Disease

Description

Targeted research and clinical trials have resulted in considerable progress in novel treatment strategies for liver disease. This session highlights innovations in both adult and pediatric hepatology that have the capacity to transform the care of pediatric patients with a spectrum of liver disease. Topics include: (1) the current landscape of ileal bile acid transporter (IBAT) inhibitors for the treatment of children with Alagille syndrome and progressive familial intrahepatic cholestasis, and investigation in other cholestatic liver diseases; (2) utilization of existing and novel therapies for cures or bridging to transplant in pediatric acute liver failure, including terlipressin, bioartificial liver systems, and hepatocyte and auxiliary transplant; (3) use of gene therapy in pediatric liver disease and the landscape for future innovations; (4) overview of current and future therapeutics for metabolic dysfunction-associated steatotic liver disease and applicability to young adults treated at pediatric centers; and (5) promising data on small interfering RNA therapy for alpha-1-antitrypsin disease and other applications.