Voretigene Neparvovec Gene Therapy in Clinical Practice: Treatment of the First Two Italian Pediatric Patients. [PDF]
Transl Vis Sci Technol, 2021 Purpose: To present visual outcomes of the first two Italian patients with RPE65-related inherited retinal dystrophy (RPE65-IRD) treated with voretigene neparvovec (VN).
Testa F +11 more
europepmc +2 more sources
Voretigene neparvovec in RPE65-related inherited retinal dystrophy: the 1-year real-world study LIGHT. [PDF]
Eye (Lond)International audienceBackground: This retrospective real-world study evaluated the effectiveness and safety of subretinal voretigene neparvovec (VN) in French patients (six children, six adults) with inherited retinal dystrophies.Methods: Data were ...
Audo I +13 more
europepmc +2 more sources
Фармакоэкономика, 2022 Background. New pathogenetic treatment options, such as gene therapy, are now used to treat previously uncurable diseases. However, price of such treatment is high, especially in the case of orphan diseases, where costs may many-fold exceed the prices ...
N. А. Avxentyev +2 more
doaj +1 more source
Voretigene Neparvovec for the Treatment of RPE65-associated Retinal Dystrophy: Consensus and Recommendations from the Korea RPE65-IRD Consensus Paper Committee. [PDF]
Korean J Ophthalmol, 2023 Mutations in the RPE65 gene, associated with Leber congenital amaurosis, early-onset severe retinal dystrophy, and retinitis pigmentosa, gained growing attention since gene therapy for patients with RPE65-associated retinal dystrophy is available in ...
Han J +8 more
europepmc +2 more sources
Therapeutic landscape for inherited ocular diseases: current and emerging therapies
Singapore Medical Journal, 2023 Inherited ocular diseases comprise a heterogeneous group of rare and complex diseases, including inherited retinal diseases (IRDs) and inherited optic neuropathies.
Hwei Wuen Chan, Jaslyn Oh, Bart Leroy
doaj +1 more source
Classification and Growth Rate of Chorioretinal Atrophy after Voretigene Neparvovec-Rzyl for RPE65-Mediated Retinal Degeneration. [PDF]
Ophthalmol Retina, 2023 PURPOSEClassify the appearance and quantify the growth rate of chorioretinal atrophy in patients who received voretigene neparvovec-rzyl (VN) for RPE65-mediated retinal degeneration.
Bommakanti N +11 more
europepmc +2 more sources
Iatrogenic choroidal neovascularization associated with subretinal gene therapy surgery
American Journal of Ophthalmology Case Reports, 2022 Purpose: To report a case of iatrogenic choroidal neovascularization (CNV) developing one month after subretinal gene therapy surgery. Observations: A 16-year-old male with biallelic RPE65 mutation associated retinal dystrophy was treated with subretinal
Kenneth W. Price +3 more
doaj +1 more source
A cost effectiveness analysis of Voretigene Neparvovec for RPE-65 Mediated Inherited Retinal Dystrophies in Greece. [PDF]
, 2021 Οι ασθενείς και ειδικά τα παιδιά με τους γονείς τους που ζουν με μια κληρονομική νόσο του αμφιβληστροειδούς δεν είχαν μέχρι στιγμής μια αποτελεσματική θεραπεία που θα μπορούσε να αλλάξει τη ζωή τους και να δώσει ελπίδα.
Filinis Georgios +1 more
core +1 more source
Journal of Enzyme Inhibition and Medicinal Chemistry, 2022 The human retinal pigment epithelium-specific 65-kDa protein (hRPE65) plays a crucial role within the retinoid visual cycle and several mutations affecting either its expression level or its enzymatic function are associated with inherited retinal ...
Giulio Poli +7 more
doaj +1 more source
Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation [PDF]
Journal of Current Ophthalmology, 2018 Hossein Ameri
doaj +2 more sources