Rare Daily Staff

Retrophin said it has entered into a three-way Cooperative Research and Development Agreement (CRADA) with the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) and the patient advocacy foundation Alagille Syndrome Alliance to identify and develop of therapies for Alagille syndrome.

Alagille syndrome is a rare genetic disorder that can affect the liver, heart, skeleton, eyes and kidneys. Symptoms and severity of the condition can vary greatly from one person to another. Symptoms often develop during the first three months of life and include interrupted bile flow, jaundice, poor weight gain and growth, and severe itching.

The collaboration seeks to develop high-throughput screening assays and conduct hit validation in an effort to identify compounds that could be advanced in development, with the goal of delivering potentially novel therapeutic options to the ALGS patient community.

“This partnership with Retrophin and NCATS offers new hope to our patients and their families,” said Roberta Smith, president of ALGSA. “We’re excited to be an active part of this collaboration aimed at advancing meaningful efforts to find potential therapies for ALGS.”

Photo: Roberta Smith, president of ALGSA

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