Active replication — ongoing trial, registry, or meta-analysis.
Recent follow-up analyses of LUXTURNA (voretigene) are confirming the original effect size in real-world data.
TL;DR · AI-generated
Voretigene neparvovec gene replacement improved functional vision in RPE65-mediated inherited retinal dystrophy previously medically untreatable.
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Evidence stream
2 events · 1 snapshot
posterior drift
71% → 71% (0pp · 1 point)
Registry data
Apr 18, 2026
Peer-reviewed paper
Apr 18, 2026
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Source publication
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al. · The Lancet · 2017
· openalex W2736065260 · s2 e00fcd8a
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The LUXTURNA (voretigene) trial's stated primary conclusion — Subretinal AAV2-hRPE65 gene therapy improves functional vision in biallelic RPE65-associated inherited retinal dystrophy. — replicates in independent cohorts.
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