About Biliary Atresia

Biliary atresia is a rare pediatric liver disease with symptoms typically developing about two to eight weeks after birth and no approved pharmacological therapies. Damaged or absent bile ducts outside the liver result in bile and bile acids being trapped inside the liver, quickly resulting in cirrhosis, and even liver failure.

The disease impacts an estimated 15,000-20,000 people in the United States and European Union, and is the most common pediatric cholestatic liver disease and the leading cause of pediatric liver transplant across all diseases. There are no approved pharmacological treatments for biliary atresia.