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