Experimental enzyme therapy targets rare kidney-damaging cholesterol disorder
NCT ID NCT04737720
First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 22 times
Summary
This study tested an artificial enzyme called ACP-501 in one person with familial LCAT deficiency, a rare genetic condition that causes low good cholesterol and kidney failure. The participant received multiple infusions of the enzyme over weeks. Researchers measured whether the treatment raised good cholesterol levels and improved kidney function, but ongoing management is still needed.
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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.
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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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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