Could a liver drug restore color vision in rare blindness?
NCT ID NCT04041232
First seen Sep 30, 2025 · Last updated May 23, 2026 · Updated 38 times
Summary
This early-phase study tests whether an FDA-approved drug called glycerol phenylbutyrate (PBA) can improve vision in people with a rare inherited form of color blindness caused by ATF6 gene mutations. Only 2 adults with confirmed ATF6 mutations and reduced retinal function are being enrolled. Participants take PBA three times daily and undergo several eye tests over at least 3 clinic visits to measure changes in vision and retinal health.
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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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Contacts and locations
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Locations
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Columbia University Irving Medical Center
New York, New York, 10032, United States
Conditions
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