JAMA ophthalmology
Vision Outcomes in Children Treated With Voretigene Neparvovec for RPE65-Associated Retinopathy.
Dorothy A Thompson, Elisabeth De Smit, Weijen Tan, Siân E Handley, Oliver R Marmoy, Robert H Henderson
Published: 202610.1001/jamaophthalmol.2025.5505
Abstract
Importance: Voretigene neparvovec (VN) marketed as Luxturna (Novartis Europharm Ltd) is the first approved gene therapy for severe RPE65-related retinal dystrophy, showing low-luminance vision improvement in adult trials. Low-luminance tests pose cha…
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