Hope for infants with rare fatal disease: new drug targets devastating liver complication

This early-phase study tested whether an intravenous drug called adrabetadex could treat serious liver disease in infants with Niemann-Pick disease, Type C (NPC), a rare and fatal genetic disorder. The trial involved four infants under six months old who showed signs of liver problems from NPC. Researchers gave the drug twice a week for six weeks, then monthly for six months for some infants, to see if it could reduce harmful substances in the blood linked to liver damage.