Results 71 to 80 of about 2,056 (145)
Retinitis Pigmentosa: Burden of Disease and Current Unmet Needs
Clinical Ophthalmology, 2022 Nancy Cross,1 Cécile van Steen,2 Yasmina Zegaoui,1 Andrew Satherley,1 Luigi Angelillo2 1Market Access, Lightning Health, London, England, UK; 2Market Access HTA & HEOR, EMEA, Santen GmbH, Munich, Bavaria, GermanyCorrespondence: Yasmina Zegaoui, Market ...
Cross N +4 more
doaj
Toward Precision Medicine: Gene Therapy Applications in the Management of Uveal Melanoma
Cancer Reports, Volume 8, Issue 12, December 2025.ABSTRACT Background Uveal melanoma (UM) is the prevailing malignant tumor that develops within the eye in adults, and it has a bleak outlook because of the few treatment choices available and the high likelihood of returning after treatment. Currently, surgical intervention, radiation therapy, and a combination of both modalities are available ...
Alireza Azani +13 more
wiley +1 more source
Molecular Therapies for Choroideremia
, 2019 Advances in molecular research have culminated in the development of novel gene-based therapies for inherited retinal diseases. We have recently witnessed several groundbreaking clinical studies that ultimately led to approval of Luxturna, the first gene
Alun R. Barnard +2 more
core +1 more source
Gene therapy for the treatment of rethinopaties [PDF]
, 2023 Las mutaciones en un elevado número de genes son responsables de retinopatías hereditarias, una serie de trastornos que producen una discapacidad visual significativa debido a la afectación de la retina, la parte del ojo sensible a la luz localizada ...
Díaz Fernández, Pablo
core
Application of gene therapy in the treatment of blindness
, 2020 Genskom terapijom uvodi se novi, funkcionalni gen u stanicu i organizam čovjeka kako bi se nadomjestio nedostatak funkcije postojećeg, često mutiranog gena te izliječila bolest, koja je nastala kao posljedica nedostatka tog proteina.
Vrdoljak, Jelena
core +2 more sources
État des lieux des thérapies géniques en ophtalmologie
, 2023 L'objectif de cette thèse est d'offrir une vue d'ensemble sur l'état actuel des thérapies géniques en ophtalmologie. Si l'approbation du Luxturna® pour l'amaurose congénitale de Leber causée par une mutation bi-allélique du gène RPE65 a été une avancée ...
Dupuy, Victor
core
Uusien sairaalalääkkeiden arviointi [PDF]
Tämä arviointi käsittelee voretigeenineparvoveekin (Luxturna) hoidollisia ja taloudellisia vaikutuksia potilailla, joilla on näön heikentymiseen johtanut molempien alleelien RPE65-mutaatioista johtuva perinnöllinen verkkokalvorappeuma, ja joilla on ...
Grönholm, Essi, Nättinen, Janika
core
HeliyonPurpose: To report the effect of internal limiting membrane (ILM) peeling prior to Voretigen Neparvovec-ryzl (VN) subretinal injection on focal chorioretinal atrophy development in patients presenting with RPE65-mediated Leber congenital amaurosis (LCA).
Lea Dormegny +7 more
doaj +1 more source
Produtos de terapia avançada aprovados pela ANVISA
Com o advento da terapia gênica foi possível ter uma nova perspectiva sobre tratamentos de diversas doenças posto que o medicamento age em nível genético visando correção das anormalidades causadoras das enfermidades.
Fogaça, Natali Pereira
core +1 more source
LifeInherited retinal diseases (IRDs) are a clinically and genetically diverse group of progressive degenerative disorders that can result in severe visual impairment or complete blindness.
Xiaoming Gong, Richard W. Hertle
doaj +1 more source