Efficacy Outcome Measures for Clinical Trials of USH2A Caused by the Common c.2299delG Mutation

In patients with the c.2299delG mutation in USH2A, rod photoreceptors are the cells that express disease early and more aggressively than cones. Rod-based vision measurements in central or extracentral-peripheral retinal regions warrant monitoring in order to complete a clinical trial in a timely manner.