Rare Diseases: Immune Thrombocytopenia Purpura

Primary Biliary Cholangitis is rare autoimmune disease targeting the liver; it affects 133,000 people in the U.S. PBC is caused by the dysregulated immune response towards proteins of liver bile duct cells which leads to immune-mediated destruction of bile ducts. As a result, bile acids build up progressively in the liver leading to irreversible damage and scarring of the liver tissue ultimately requiring liver transplant. Currently there is no cure for PBC. Medical care is focused only on the management of symptoms and does not address the underlying dysregulated immune system responsible for disease progression or recovery of the damaged tissue.