Experimental drug targets deadly liver disease in newborns
NCT ID NCT03471143
First seen Jan 06, 2026 · Last updated May 08, 2026 · Updated 16 times
Summary
This early-phase study tested an experimental drug called adrabetadex (VTS-270) in 4 infants with Niemann-Pick type C disease who had severe liver problems. The drug was given through a vein to see if it could lower abnormal bile acid levels in the blood, which are a sign of liver damage. The study was small and open-label, meaning everyone knew they were getting the drug, so results are preliminary.
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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
Conditions
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