Alagille Syndrome

Alagille syndrome is a rare genetic disorder that can affect the liver, heart, and other parts of the body. Approximately one in every 70,000 children is born with Alagille syndrome. In patients with Alagille syndrome, the bile ducts are abnormally narrow, malformed and reduced in number. This prevents the bile ducts from carrying bile, which helps to digest fats, from the liver to the gallbladder and small intestine. As a result, bile builds up in the liver, causing scarring that prevents the liver from working properly to eliminate wastes from the body. Inhibiting the ASBT, which is responsible for recycling bile acids back to the liver, may mimic the effects of successful surgical approaches that reduce bile accumulation in the liver, thereby improving liver function and symptoms of disease. 

Lumena is currently conducting a Phase II clinical study of LUM001 in children with Alagille syndrome | More Information »

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