Treating Progressive Familial Intrahepatic Cholestasis (PFIC)

摘要

Provided herein are methods for treating progressive familial intrahepatic cholestasis (PFIC) with an ileal bile acid transport (IBAT) inhibitor such as odevixibat, or a pharmaceutically acceptable salt thereof. Such methods can include reducing mean pruritus score, mean serum bile acid concentration, increasing height, normalizing weight, improving sleep, and improving liver parameters.