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Announcements Niemann-Pick Type C News Research News

IV Adrabetadex (cyclodextrin) Trial for Neonatal Liver Disease Started

Researchers from St. Louis Children’s Hospital at Washington University School of Medicine are enrolling infants 0-6 months with diagnosed NPC disease and evidence of liver disease.  The purpose of the study is to learn how well adrabetadex can reduce liver damage in infants diagnosed with NPC disease. The study has an initial 6-week phase during …