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Article

Real-world outcomes of Voretigene Neparvovec: a single-centre consecutive case series.

Eye (London, England) - 1 May 2025

Jalil Assad, Ferrara Mariantonia, Lippera Myrta, Parry Neil, Black Graeme C, Banderas Sandra, Ashworth Jane, Biswas Sus, Hall Georgina, Gray Jane, Newman William, Ivanova Tsveta

Abstract excerpt

PURPOSE: To present functional and anatomical outcomes of subretinal therapy with Voretigene Neparvovec (VN) in patients treated in one of the four specialist UK gene therapy centres. METHODS: Single-centre, retrospective case series of patients affected by an inherited retinal dystrophy (IRD) caused by a pathogenic biallelic RPE65 mutation and treated with VN. Complete ophthalmic examination was planned...

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Identifiers

PMID: 39900645
DOI: 10.1038/s41433-025-03637-0
PMCID: PMC12043895

Genes

RPE65

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