Ontology highlight
ABSTRACT:
SUBMITTER: Heubi JE
PROVIDER: S-EPMC5559188 | biostudies-literature | 2017 Sep
REPOSITORIES: biostudies-literature
Heubi James E JE Bove Kevin E KE Setchell Kenneth D R KDR
Journal of pediatric gastroenterology and nutrition 20170901 3
<h4>Objectives</h4>Patients with bile acid synthesis disorders (BASDs) due to single enzyme defects (SEDs) or Zellweger spectrum disorders (ZSDs) accumulate hepatotoxic atypical bile acids resulting in potentially fatal progressive liver disease. We evaluated the efficacy and safety of oral cholic acid in patients with BASD.<h4>Methods</h4>In this phase 3, open-label, single-arm, nonrandomized, noncomparative study conducted over 18 years, patients were administered cholic acid orally 10 to 15 m ...[more]