Inherited Retinal Dystrophy Due to Rpe65 Mutations
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CLINICAL_TRIALS trial_result · 2025-04-18Clinical Trial Update: Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)
This clinical trial update presents long-term follow-up data from subjects treated with voretigene neparvovec-rzyl (AAV2-hRPE65v2) for inher…
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CLINICAL_TRIALS trial_result · 2018-03-26Clinical Trial Update: Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis [Phase 3]
A Phase 3 study evaluating safety and efficacy in subjects with Leber congenital amaurosis due to RPE65 mutations has posted results.