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 safety check: luxturna registry wraps up

Knowledge-focused Completed

This completed study tracked 87 patients who received Luxturna gene therapy for a rare inherited eye condition that causes blindness. Researchers monitored side effects and pregnancy outcomes for up to 5 years after treatment. The goal was to gather real-world safety data, not to…

Matched conditions: CONFIRMED BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY

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

Last updated Jun 27, 2026 07:51 UTC

Confirmed biallelic RPE65 mutation-associated retinal dystrophy

Get alerted when new Confirmed biallelic RPE65 mutation-associated retinal dystrophy trials appear

Gene therapy safety check: luxturna registry wraps up