Article
Current Management of Patients with RPE65 Mutation Associated Inherited Retinal Degenerations in Europe: Results of a 2-Year Follow-Up Multinational Survey.
Ophthalmic research - 1 Jan 2023
Lorenz Birgit, Tavares Joana, van den Born L Ingeborgh, Marques João Pedro, Pilotto Elisabetta, Stingl Katarina, Charbel Issa Peter, Leroux Dorothée, Dollfus Hélène, Scholl Hendrik P N
Abstract excerpt
INTRODUCTION: The aim of this study was to evaluate the current management of RPE65 biallelic mutation-associated inherited retinal degeneration (RPE65-IRD) in Europe since market authorization of voretigene neparvovec (VN, LuxturnaTM) in 2018. By July 2022, over 200 patients have been treated outside the USA, of whom about 90% in Europe. We conducted among all centers of the European Vision Institute Clinical...
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