congenital bile acid synthesis defect 3

MeSH: C566340ORPHA: 79302

Overview

congenital bile acid synthesis defect characterized by intrahepatic cholestasis, malabsorption of fat and fat-soluble vitamins, and increased serum bilirubin that has material basis in homozygous mutation in the CYP7B1 gene on chromosome 8q12

Available Treatments (0)

No treatments linked yet

Clinical trials or compassionate use may be available — consult a specialist.

Clinical Presentation

Signs and symptoms associated with congenital bile acid synthesis defect 3, sourced from HPO and Orphanet clinical annotations.

JaundiceCirrhosisCholestasisHepatic failureBile duct proliferationHepatosplenomegalyFat malabsorptionHyperbilirubinemiaElevated circulating alkaline phosphatase concentrationElevated circulating aspartate aminotransferase concentrationElevated circulating alanine aminotransferase concentrationAbnormality of vitamin metabolism

Classification & Codes

MeSH Code

C566340

Orphanet Code

ORPHA:79302
congenital bile acid synthesis defect 3
MeSHC566340
OrphanetORPHA:79302
Treatments0 drug(s)
Symptoms on record12 signs
Statuspublished
Factual Authority
Last Updated3/20/2026
Clinical DataHPO · Orphanet
Drug DataFDA · EMA · CDSCO