2015 Research Annual Report

Pathology and Laboratory Medicine

Glycocholic Acid Proves Effective Against Newly-Identified Amidation Defect

Over the past 30 years, James E. Heubi, MD, and Kenneth D. Setchell, PhD, have revolutionized the treatment of liver disease in children, and their novel findings led to the March 2015 FDA approval of the drug Cholbam. This bile acid treatment – when given to children – tricks the liver into thinking it is producing enough of its own healthy bile acid so that it shuts down production of defective bile acids that lead to liver disease.