Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence
Sign Up to like & getrecommendations! Published in 2022 at "Molecular Genetics & Genomic Medicine"
DOI: 10.1002/mgg3.2038
Abstract: Leber congenital amaurosis (LCA) type 2, due to disease‐causing variants in RPE65, is characterized by severe visual loss in early infancy. Current treatments include voretigene neparvovec‐rzyl (VN) for RPE65‐associated LCA. Herein, we present the long‐term… read more here.
Keywords: atrophy following; chorioretinal atrophy; voretigene neparvovec; autofluorescence ... See more keywords