Results 1 to 10 of about 1,195 (111)

Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence [PDF]

Molecular Genetics & Genomic Medicine, 2022
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.
Janet R Sparrow, Stephen Tsang, Vinit B Mahajan   +2 more
exaly   +3 more sources

Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study [PDF]

Biomolecules
Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic RPE65-mediated inherited retinal dystrophy who have sufficient viable retinal cells. PERCEIVE is an ongoing, post-authorization, prospective, multicenter, registry-
Francesca Simonelli, Jayashree Sahni, Bart Leroy   +2 more
exaly   +4 more sources

Bilateral exudative retinal detachments after subretinal gene therapy with voretigene neparvovec-rzyl for RPE65 Leber Congenital Amaurosis [PDF]

American Journal of Ophthalmology Case Reports, 2023
Purpose: To report panuveitis with exudative retinal detachments in a healthy 27-year-old woman with biallelic mutations in the RPE65 gene, who underwent bilateral sequential gene therapy with subretinal administration of voretigene neparvovec-rzyl ...
Alcina K. Lidder, Stephanie Choi, Yasha S. Modi, Scott E. Brodie, Janet L. Davis, Ninel Z. Gregori, Byron L. Lam   +6 more
doaj   +2 more sources

Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis [PDF]

Biomedicines, 2022
Leber congenital amaurosis caused by mutations in the RPE65 gene belongs to the most severe early-onset hereditary childhood retinopathies naturally progressing to legal blindness.
Maximilian J. Gerhardt, Claudia S. Priglinger, Günther Rudolph, Karsten Hufendiek, Carsten Framme, Herbert Jägle, Daniel J. Salchow, Andreas Anschütz, Stylianos Michalakis, Siegfried G. Priglinger   +9 more
doaj   +2 more sources

Sustained improvement in dark-adapted sensitivity but not BCVA after voretigene neparvovec treatment in a mainland Chinese child with biallelic RPE65-associated LCA2: a case report [PDF]

Frontiers in Pharmacology
Inherited retinal dystrophies (IRDs) represent heterogeneous genetic eye disorders frequently linked to RPE65 mutations. Voretigene neparvovec (VN, Luxturna), an AAV2-mediated gene therapy, remains the sole approved drug for biallelic RPE65-associated ...
Yue Ren, Yue Ren, Shu Liu, Yanbo Liu, Di Wang, Jia Rong, Wensheng Li, Chunhui Zheng, Ling Xu, Jun Li, Zhuoshi Wang, Jijing Pang, Jijing Pang   +12 more
doaj   +2 more sources

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

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 ...
Peter Kiraly, Charles L. Cottriall, Laura J. Taylor, Jasleen K. Jolly, Jasmina Cehajic-Kapetanovic, Imran H. Yusuf, Cristina Martinez-Fernandez de la Camara, Morag Shanks, Susan M. Downes, Robert E. MacLaren, M. Dominik Fischer   +10 more
doaj   +2 more sources

Assessing the Accuracy, Quality, and Readability of Patient Accessible Online Resources Regarding Ocular Gene Therapy and Voretigene Neparvovec [PDF]

Clinical Ophthalmology, 2021
Swetha Davuluri,1 Nicolas A Yannuzzi,1 Amy Kloosterboer,1 Ajay E Kuriyan,2 Jayanth Sridhar1 1Department of Ophthalmology, Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami, FL, USA; 2Department of Retina Service, Mid ...
Davuluri S, Yannuzzi NA, Kloosterboer A, Kuriyan AE, Sridhar J   +4 more
doaj   +2 more sources

Cost-effectiveness of voretigene neparvovec in the treatment of patients with inherited retinal disease with RPE65 mutation in Switzerland [PDF]

BMC Health Services Research, 2022
Objective We aimed to evaluate the cost-effectiveness of voretigene neparvovec (VN) compared with standard of care (SoC) for patients with inherited retinal disease (IRD) caused by a biallelic RPE65-mutation.
Arjun Bhadhuri, Daniel Dröschel, Mike Guldimann, Claudia Jetschgo, Judit Banhazi, Matthias Schwenkglenks, C. Simone Sutherland   +6 more
doaj   +2 more sources

Visual function and retinal changes after voretigene neparvovec treatment in children with biallelic RPE65-related inherited retinal dystrophy [PDF]

Scientific Reports, 2022
To report quantitative retinal changes assessed by spectral-domain optical coherence tomography (SD-OCT) in children treated with voretigene neparvovec (VN) at a single center in Italy.
Francesco Testa, Paolo Melillo, Valentina Di Iorio, Claudio Iovino, Francesco Farinaro, Marianthi Karali, Sandro Banfi, Settimio Rossi, Michele Della Corte, Francesca Simonelli   +9 more
doaj   +2 more sources

Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence [PDF]

Clinical Ophthalmology, 2020
Jie Gao,1 Rehan M Hussain,2 Christina Y Weng1 1Department of Ophthalmology, Baylor College of Medicine, Houston, TX, USA; 2Retina Associates, Elmhurst, IL, USACorrespondence: Christina Y WengBaylor College of Medicine, Alkek Eye Center, 1977 Butler Blvd,
Gao J, Hussain RM, Weng CY
doaj   +2 more sources