To design a good clinical trial, scientists need to understand how a disease changes over time and how to measure whether a treatment is working. In this study, researchers wanted to learn more about how USH2A affects people and which vision tests are best to track changes.
Researchers from 16 locations in Europe and North America worked together. They used simple, non-invasive tools to check the eye health and vision of 105 people with USH2A. After looking at all the results, they made suggestions for how to design future clinical trials to test new treatments for USH2A.
What this means for Usher syndrome: The information from this study will help researchers plan future clinical trials for USH2A treatments. It will also help doctors know what to measure to determine whether a treatment is working.
