First-ever single-patient trial targets rare blindness and balance disorder
NCT ID NCT06565572
First seen May 20, 2026 · Last updated May 20, 2026
Summary
This early-stage study tests a new type of genetic medicine (called an antisense oligonucleotide) in just one person with PCARP, a rare disease that causes vision loss and trouble with balance. The main goal is to see if the treatment is safe and tolerable. The participant will receive an injection into the eye, and doctors will monitor for side effects and any changes in vision.
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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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Childrens Hospital Colorado
Aurora, Colorado, 80045, United States
Conditions
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