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.
There are currently no approved pharmacological treatments for biliary atresia, which impacts an estimated 40,000 to 50,000 people worldwide.
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