Catherine Cibulskis, MD – Cholestasis

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This is a compassionate use protocol of an investigational new drug (IND). The overall
purpose of the treatment is to offer alternative treatment to children who developed
parenteral nutrition-associated liver disease (PNALD) and have not responded positively to
currently available medical therapies. PNALD develops in newborns dependent on parenteral
nutrition (PN) and are unable to tolerate adequate enteral feedings to support fluid and
nutritional fluids; although PN is necessary and life sustaining, it can result in severe
liver disease.

Rare Disease: