CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY

Clinical trials for CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY explained in plain language.

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Completed 1

Gene therapy for rare blindness passes 5-year safety check

Knowledge-focused Completed

This study followed 87 people who had already received Luxturna gene therapy for a rare inherited eye condition that can cause blindness. The goal was to watch for any side effects over 5 years after treatment. No new treatment was given; researchers simply collected safety infor…

Matched conditions: CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY

Sponsor: Spark Therapeutics, Inc. • Aim: Knowledge-focused

Last updated May 18, 2026 03:48 UTC

CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY

Get alerted when new CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY trials appear

Gene therapy for rare blindness passes 5-year safety check