BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases

Inclusion Criteria: * with signed informed consent form from the guardian, and the patient if applicable. * aged from 2 month to 18 years old. * with cholestatic disease caused by ABCB11 biallelic mutation. * Long-term residence in China. Exclusion Criteria: * Currently receiving or previously received experimental drugs. * The child is already in the stage of liver failure, or in unstable state that are not suitable for drug treatment according to the researcher's judgment: serious complications such as bleeding tendency and skin rash. * accompany with other chronic liver disease (viral hepatitis B and C, autoimmune hepatitis, wilson disease, cystic fibrosis, primary biliary cirrhosis, biliary atresia, sclerosing cholangitis, bile acid synthesis defects, and infections, cholestasis caused by space-occupying and other reasons). * Suffered from congenital TORCHES infection, including toxoplasma gondii, rubella virus, cytomegalovirus, herpes simplex virus, EB virus, syphilis, HIV, etc. * With any other major medical conditions that may affect drug absorption, metabolism, or excretion based on the researcher's judgment. * Known or suspected hypersensitivity to any experimental drugs or their indigents. * Patients with alcohol or drug dependence. * In receiving any investigational drugs or within 60 days before enrollment.