Results 11 to 20 of about 1,367 (120)

Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis [PDF]

Ophthalmology Retina, 2022
PURPOSE: To report an anatomic change observed in a series of patients who underwent subretinal injection of voretigene neparvovec-rzyl (VN) for RPE65-mediated Leber congenital amaurosis. DESIGN: Multi-center retrospective chart review.
Çağrı G. Beşirli, Aaron Nagiel, William S Gange   +2 more
exaly   +3 more sources

Gene therapies in pediatric ophthalmology [PDF]

Frontiers in Ophthalmology, 2023
Genetic pediatric eye disease frequently leads to severe vision impairment or blindness. Voretigene neparvovec is the first approved gene therapy for an inherited retinal dystrophy (IRD). Voretigene neparvovec has been shown to be well tolerated and safe,
Alejandra Daruich, Alejandra Daruich, Matthieu P. Robert, Matthieu P. Robert, Dominique Bremond-Gignac, Dominique Bremond-Gignac   +5 more
doaj   +3 more sources

Gene therapy and hospital pharmacy: the experience of the Hospital Pharmacy unit of L. Vanvitelli university hospital [PDF]

AboutOpen, 2021
Background: Since hospital use of gene therapy requires close cooperation between doctors and pharmacists to ensure correct drug handling and administration, it is essential to define standard operating procedures (SOPs) for each phase of the process to ...
Giovanni Dubuis, Francesca Marrone, Francesco Russo, Stefania Ziccardi, Rosa Annibale   +4 more
doaj   +2 more sources

Improvements induced by retinal gene therapy with voretigene neparvovec depend on visual cortical hemispheric dominance mechanisms [PDF]

Communications Medicine
Background RPE65-associated retinal degeneration (RPE65-RD) causes severe visual deficits. Gene therapy with AAV2-hRPE65v2 is a breakthrough but it is currently unknown which visual pathways benefit from treatment and if cortical mechanisms can amplify ...
Mariana Ferreira, João Pedro Marques, Miguel Raimundo, Hugo Quental, Miguel Castelo-Branco   +4 more
doaj   +2 more sources

Photopharmacology: A new paradigm for vision restoration. [PDF]

Clin Transl Med
Clinical and Translational Medicine, Volume 16, Issue 8, August 2026.
Casson RJ.
europepmc   +2 more sources

Full-field Scotopic Threshold Improvement after Voretigene Neparvovec-rzyl Treatment Correlates with Chorioretinal Atrophy [PDF]

Ophthalmology, 2023
Aaron Nagiel, Krunoslav Stingl, Friederike Kortuem   +2 more
exaly   +2 more sources

Short-Term Outcomes of Pediatric Patients With Mild Autosomal Recessive RPE65-Associated Retinal Dystrophy Treated With Voretigene Neparvovec. [PDF]

Transl Vis Sci Technol
PURPOSE: Voretigene neparvovec is approved for RPE65-associated inherited retinal degeneration (RPE65-IRD) in the United States and the European Union. According to current knowledge, early treatment benefits efficacy. However, consensus on treating mild
Merle DA, Hertens L, Dimopoulos S, Kohl S, Van Haute M, De Baere E, De Bruyne M, Janssens B, Rüther K, Huchzermeyer C, Mazzola P, Nerinckx F, Haack T, Wolfram L, Kempf M, Kühlewein L, Stingl K, Leroy BP, Stingl K.   +18 more
europepmc   +2 more sources

Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec. [PDF]

Br J Ophthalmol, 2022
BACKGROUND: Voretigene neparvovec is a gene therapeutic agent for treatment of retinal dystrophies caused by bi-allelic RPE65 mutations. In this study, we report on a novel and objective evaluation of a retinotopic photoreceptor rescue.
Stingl K, Kempf M, Bartz-Schmidt KU, Dimopoulos S, Reichel F, Jung R, Kelbsch C, Kohl S, Kortüm FC, Nasser F, Peters T, Wilhelm B, Wissinger B, Wozar F, Zrenner E, Fischer MD, Stingl K.   +16 more
europepmc   +2 more sources

12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65-mediated inherited retinal dystrophy. [PDF]

Br J Ophthalmol
AIMS: To report main outcomes and complications following voretigene neparvovec (Luxturna) treatment in paediatric patients. METHODS: Records of patients under the age of 17 treated by subretinal administration of voretigene neparvovec for confirmed ...
Daruich A, Rateaux M, Batté E, de Vergnes N, Valleix S, Robert MP, Bremond Gignac D.   +6 more
europepmc   +2 more sources

Perimacular Atrophy Following Voretigene Neparvovec-Rzyl Treatment in the Setting of Previous Contralateral Eye Treatment With a Different Viral Vector. [PDF]

Transl Vis Sci Technol
Purpose: To report on cases of unilateral perimacular atrophy after treatment with voretigene neparvovec-rzyl, in the setting of previous contralateral eye treatment with a different viral vector.
Ku CA, Igelman AD, Huang SJ, Bailey ST, Lauer AK, Duncan JL, Weleber RG, Yang P, Pennesi ME.   +8 more
europepmc   +2 more sources