The RUSH2A Natural History Study follows people who have changes in the USH2A gene that cause Usher syndrome type 2A or non-syndromic retinitis pigmentosa (RP). The study tracks these individuals for several years to see how their condition naturally progresses without treatment. By learning how the disease changes over time, scientists can design better clinical trials and develop more effective treatments.
So far, RUSH2A has gathered four years of data from more than 100 people. The Foundation Fighting Blindness is now sharing this information publicly, with all personal details removed. By making the data available, researchers worldwide can use them to develop better studies and endpoints for future clinical trials.
The study will run for a total of nine years and uses tests like visual acuity, light sensitivity, and eye imaging to track how vision changes over time. All study sites follow the same procedures, helping keep the data consistent and reliable. The Foundation has invested over $3 million in RUSH2A, with support from partner companies. Researchers have already published many papers using the data, and even more findings are expected as the study continues.
What this means for the Usher syndrome community: The RUSH2A study is especially valuable because it shows how vision naturally changes in people with USH2A-related conditions, including Usher syndrome type 2A. These insights will guide companies in their research as they work to develop new treatments.
