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
Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation
Sign Up to like & getrecommendations! Published in 2018 at "Journal of Current Ophthalmology"
DOI: 10.1016/j.joco.2018.01.006
Abstract: The approval of voretigene neparvovec-rzyl by the US Food and Drug Administration (FDA), in December 2017, marked the beginning of a new era in medicine in which many inherited diseases will be essentially corrected by… read more here.
Keywords: voretigene neparvovec; gene; neparvovec rzyl; gene therapy ... See more keywords
Subretinal deposits in young patients treated with voretigene neparvovec-rzyl for RPE65-mediated retinal dystrophy
Sign Up to like & getrecommendations! Published in 2022 at "British Journal of Ophthalmology"
DOI: 10.1136/bjo-2022-321488
Abstract: We report a series of three young patients (ages: 22 months, 2 years, and 5 years) who developed subretinal deposits at post-operative week one following subretinal voretigene neparvovec-rzyl treatment for RPE65-mediated retinal dystrophy. In the… read more here.
Keywords: subretinal deposits; neparvovec rzyl; rpe65 mediated; mediated retinal ... See more keywords
Gene therapy for inherited retinal disease: long-term durability of effect.
Sign Up to like & getrecommendations! Published in 2022 at "Ophthalmic research"
DOI: 10.1159/000526317
Abstract: The recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65 mutation-associated inherited retinal dystrophy with viable retinal cells represents an important step in the development of ocular gene therapies. Herein, we review studies… read more here.
Keywords: long term; disease; voretigene neparvovec; inherited retinal ... See more keywords