We have a path to clinical trials for those with Usher Syndrome.
Over $4.5 million and a decade of work has brought us to the first Usher 2020-supported clinical trial, but that is only the beginning.
What Have We Done?
Usher 2020 has spent years funding cutting-edge research in small molecule therapy, gene therapy, and stem cell therapy. We are proud to have partnered with some of the most respected and experienced clinicians and researchers in the field of rare inherited retinal diseases like Usher Syndrome.
What Will We Do Now?
Beginning in early 2023, work funded by Usher 2020 out of The Wilmer Eye Institute, Johns Hopkins University, will begin recruiting patients with Retinitis Pigmentosa, including those with Usher syndrome, to a 4 year, multi-location, international study of the drug N-Acetylecystine and its ability to slow the progression of vision loss caused by RP. But that is not all.
Our gene therapy treatment for the USH 1C mutation is currently being tested for efficacy in a large animal model, and we are aggressively pushing to move this treatment into a clinical trial by 2025.
Usher 2020 Foundation is proud to support the NAC Attack Clinical Study for Retinitis Pigmentosa.