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12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65 -mediated inherited retinal dystrophy.
Daruich Alejandra, Rateaux Maxence, Batté Emilie, de Vergnes Nathalie, Valleix Sophie, Robert Matthieu P et al.
Br J OphthalmolJan 202510 citationsCase Series
Pediatric RPE65 gene therapy (Luxturna) significantly improved visual acuity at 12 months. Despite some complications, these gains in visual function were maintained, highlighting its clinical benefit.