Could an existing drug restore color vision in rare blindness?
NCT ID NCT04041232
First seen Sep 30, 2025 · Last updated May 13, 2026 · Updated 34 times
Summary
This early-phase study tests whether glycerol phenylbutyrate (PBA), a drug already approved for other conditions, can improve vision in people with a rare inherited form of color blindness (achromatopsia) caused by ATF6 gene mutations. Two adults with the condition will take PBA three times daily and undergo several eye tests over three clinic visits. The goal is to see if the drug can reduce stress in the retina and improve color vision and other visual functions.
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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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Columbia University Irving Medical Center
New York, New York, 10032, United States
Conditions
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