Travere Therapeutics is participating in a three-way Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health (NIH) National Center for Advancing Translational Sciences (NCATS) and patient advocacy foundation Alagille Syndrome Alliance (ALGSA) to collaborate on research efforts aimed at the identification and development of potentially novel therapeutics for Alagille syndrome (ALGS). ALGS is a rare and debilitating disease characterized by severe liver and cardiovascular abnormalities.
ALGS is a rare genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. Symptoms often develop during the first three months of life and include interrupted bile flow (cholestasis), jaundice, poor weight gain and growth, and severe itching (pruritis). There are no approved therapies for ALGS. Learn more about ALGS.
The program goals are to develop high-throughput screening assays and conduct hit validation to identify potential new compounds that could be advanced in development, with the goal of delivering new and potentially novel therapeutic options to the ALGS patient community.