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Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence

INTRODUCTION: 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 follow‐up of a patient treat...

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Detalles Bibliográficos
Autores principales: Kolesnikova, Masha, Lima de Carvalho, Jose Ronaldo, Parmann, Rait, Kim, Angela H., Mahajan, Vinit B., Tsang, Stephen H., Sparrow, Janet R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9651599/
https://www.ncbi.nlm.nih.gov/pubmed/36225124
http://dx.doi.org/10.1002/mgg3.2038