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Gene therapy for rare Blindness-Deafness condition passes safety check in small study

NCT ID NCT02065011

First seen Jan 21, 2026 · Last updated May 23, 2026 · Updated 24 times

Summary

This study looks at the long-term safety of a gene therapy called SAR421869 for people with Usher syndrome type 1B, a rare condition that causes vision and hearing loss. Nine participants who received the treatment in a previous study are being followed for side effects and signs of biological activity. The goal is to see if the therapy is safe over time and whether it can slow retinal damage.

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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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Contacts and locations

Locations

  • Investigational Site Number : 250001

    Paris, 75012, France

  • Oregon Health and Science University Site Number : 840001

    Portland, Oregon, 97239-3098, United States

Conditions

Explore the condition pages connected to this study.