Hope on the Horizon: Breakthrough Phase 2 Results for Stargardt Disease Therapy

At A Race Against Blindness, we’re always watching for scientific breakthroughs that could bring new hope to families living with rare retinal diseases. One such development was just announced by Nanoscope Therapeutics , and it’s a milestone worth sharing. Nanoscope has published the results of their STARLIGHT Phase 2 clinical trial in eClinical Medicine (part of The Lancet Discovery Science ), marking the first and only clinical trial to date to demonstrate vision improvements in people with Stargardt disease — a genetic eye condition that currently has no approved treatment. What is Stargardt Disease? Stargardt disease, sometimes called juvenile macular dystrophy, affects more than 40,000 people in the U.S. and can leave over 20,000 legally blind. It causes the gradual loss of central vision when light-sensing photoreceptor cells in the macula break down. For many, vision loss begins in childhood or adolescence, and until now, options for treatment have been non-existent. Inside the STARLIGHT Trial Study design: Phase 2, open-label study Six participants with severe vision loss fro