Nacuity Pharmaceuticals has shared exciting news about a potential new treatment for people with Usher syndrome. They tested an antioxidant tablet called NPI-001 to see if it could slow down vision loss. In this trial, 49 people with Usher syndrome in Australia participated. Half received the antioxidant tablet, and half received a fake pill (placebo) twice a day for two years. Doctors carefully tracked changes in their vision and the health of their retina and the results were encouraging: people who took the NPI-001 tablet had a much slower rate of photoreceptor loss compared to people who took the placebo. Based on these positive results, Nacuity plans to start a larger study in 2026 to confirm these findings.
The NPI-001 tablet contains an antioxidant called NACA. NACA is very similar to another antioxidant called NAC which is being tested in the "NAC Attack" trial that recently finished enrolling participants in the US, Canada, and Europe. This is promising news for the Usher syndrome community, because NPI-001 and other antioxidant therapies have the potential to help all people with Usher syndrome, regardless of subtype.
