Results 101 to 110 of about 1,367 (120)

Phase I/IIa Study of an Intravitreal Optogenetic Therapy (AGN-151597) in Patients with Advanced Retinitis Pigmentosa. [PDF]

Ophthalmol Sci
Bunnell B, López FJ, Yuan D, Robinson MR, Duncan JL, Birch DG.   +5 more
europepmc   +1 more source

Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center

Biomolecules, 2023
Our study evaluated the morphological and functional outcomes, and the side effects, of voretigene neparvovec (VN) gene therapy for RPE65-mediated inherited retinal dystrophies (IRDs) in 12 eyes (six patients) at the Oxford Eye Hospital with a mean ...
Susan M Downes, Jasleen K Jolly, Imran Yusuf   +2 more
exaly   +8 more sources

Inflammation after Voretigene Neparvovec Administration in Patients with RPE65-Related Retinal Dystrophy

Ophthalmology, 2022
Purpose: To report on the prevalence of intraocular inflammation after subretinal voretigene neparvovec (VN) administration. Design: Retrospective review of medical files. Participants: All patients receiving VN in Denmark.
Line Kessel, Kristian Klemp
exaly   +2 more sources

Voretigene neparvovec-rzyl for treatment of RPE65-mediated inherited retinal diseases: a model for ocular gene therapy development

Expert Opinion on Biological Therapy, 2020
Introduction: Over a decade of research and development culminated in the 2017 United States (US) Food and Drug Administration (FDA) approval of voretigene neparvovec-rzyl (VN) for RPE65 mutation-associated inherited retinal disease (IRD), the first ...
Thomas A Ciulla, Aaron Nagiel, Audina M Berrocal   +2 more
exaly   +2 more sources

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Lancet, The, 2017
Background: Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy ...
Leslie Raffini, Francesca Simonelli, Chris A. Johnson   +2 more
exaly   +3 more sources