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Article

12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65 -mediated inherited retinal dystrophy

21 Nov 2024

Abstract excerpt

AIMS: To report main outcomes and complications following voretigene neparvovec (Luxturna) treatment in paediatric patients. METHODS: -mediated inherited retinal dystrophy were retrospectively reviewed. Best-corrected visual acuity (BCVA) and data from spectral-domain optical coherence tomography, ultra-wide-field fundus imaging and Goldmann visual field (VF) were analysed at 12 months follow-up. RESULTS: 12 eyes...

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PMID: 39578019
DOI: 10.1136/bjo-2024-326221

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