Rate of Progression of PCDH15-Related Retinal Degeneration in Usher

RUSH1F

Summary

This natural history study of patients with PCDH15 disease-causing variants will accelerate the development of outcome measures for clinical trials. Sensitive, reliable outcome measures of retinal degeneration will greatly facilitate development of treatments for retinitis pigmentosa due to PCDH15 disease-causing variants. Together these approaches are expected to have an impact on understanding PCDH15 related retinal degeneration, developing experimental treatment protocols, and assessing their effectiveness.

Design

Multicenter, longitudinal, prospective natural history study. Participants will be assigned to one of two Vision Cohorts based on visual acuity (VA) and kinetic visual fields (VF). Approx. 10 sites.