Experimental drug targets liver disease in rare, fatal infant disorder
NCT ID NCT03471143
First seen Jan 06, 2026 · Last updated May 22, 2026 · Updated 19 times
Summary
This early-phase study tested a drug called adrabetadex in 4 infants with Niemann-Pick disease type C (NPC), a rare and fatal genetic disorder. The drug was given through a vein to see if it could help treat liver problems that often occur in newborns with NPC. The study measured changes in bile acid levels in the blood as a sign of liver health.
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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Locations
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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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