New drug aims to save sight in rare genetic disorder

NCT ID NCT07290530

First seen Jan 07, 2026 · Last updated May 16, 2026 · Updated 24 times

Summary

This study tests whether an experimental drug called NPI-001 can slow the loss of light-sensing cells in the eyes of people with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 adults will take either the drug or a placebo twice daily for two years. Researchers will measure changes in the area of healthy retina to see if the drug preserves vision.

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