Bile acid synthesis disorders are rare congenital diseases which, if untreated, can lead to cirrhosis and end-stage liver disease. Cholic acid administration is the only treatment which can prevent patients from fatal outcomes. Since 2013 in Europe there is just one formulation of cholic acid: Orphacol®. It is difficult to administer to infant patients because of its formulation (capsules) and the need for dose titration in relation to the patient's weight.
Safety and effectiveness of compounded galenic cholic acid for bile acid synthesis disorder: a case report
Burlo, Francesca;Maggiore, Giuseppe;Barbi, Egidio
2022-01-01
Abstract
Bile acid synthesis disorders are rare congenital diseases which, if untreated, can lead to cirrhosis and end-stage liver disease. Cholic acid administration is the only treatment which can prevent patients from fatal outcomes. Since 2013 in Europe there is just one formulation of cholic acid: Orphacol®. It is difficult to administer to infant patients because of its formulation (capsules) and the need for dose titration in relation to the patient's weight.File in questo prodotto:
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