Safety and Effectiveness of Compounded Galenic Cholic Acid for Bile Acid Synthesis Disorder: A Case Report.

Background: Bile acid synthesis disorders are rare congenital diseases that can lead to cirrhosis and end-stage liver disease if left untreated. Cholic acid administration is the only treatment that can prevent patients from fatal outcomes. Since 2013 in Europe, there has been just one formulation of cholic acid: Orphacol®.