Mapping the slow fade of RPE65 blindness to speed future cures
Knowledge-focused
Completed
Researchers are following people with Leber congenital amaurosis caused by RPE65 gene mutations to see how their vision and retinas change over time. The study enrolls children and adults, ages 3 and older, who have this inherited retinal dystrophy. Participants undergo regular e…
Sponsor: MeiraGTx UK II Ltd • Aim: Knowledge-focused
Last updated Sep 21, 2026 20:00 UTC