Cholestatic and Genetic Liver Diseases: Management Across the Lifespan, Part 1

Description

Major advances have been made over the past decade in the genetic diagnosis and management of cholestatic liver disease in childhood. Adult hepatologists are now caring for increasing numbers of these patients who are receiving new medications, including ileal bile acid transporter (IBAT) inhibitors and the highly effective cystic fibrosis transmembrane regulator (CFTR) modulators. Ongoing clinical trials are also evaluating gene-based therapy for some of these diseases. Emerging data suggest that these novel therapies may favorably alter the natural history of cholestatic and genetic liver diseases. Part 1 of the cholestatic and genetic liver disease program specifically addresses cholestatic liver diseases of genetic etiology, including Alagille syndrome and progressive familial intrahepatic cholestasis.