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Description

With regular treatments, the benefits build up

Methods: Patients within the Childhood Liver Disease Research Network (ChiLDReN) enrolled in the Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) study were identified with BASD by local centers via biochemical evidence based on mass spectrometry analysis of urine/serum or from biallelic pathologic mutations in genes encoding enzymes in the bile acid synthetic pathway