JAMA ophthalmology۲۰۲۶نمایه شده در PubMed
Dorothy A Thompson, Elisabeth De Smit, Weijen Tan, Siân E Handley, Oliver R Marmoy, Robert H Henderson
چکیده
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 challenges for children, but pattern visual evoked potentials…
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DOI: 10.1001/jamaophthalmol.2025.5505