1. Voretigene neparvovec for biallelic RPE65-mediated retinal disease: effectiveness and value : final evidence report Publication: [Boston, Massachusetts] : Institute for Clinical and Economic Review, February 14, 2018 Subject(s): Genetic TherapyLeber Congenital Amaurosis -- geneticsLeber Congenital Amaurosis -- therapyRetinal Diseases -- geneticsRetinal Diseases -- therapyRetinal Pigment Epithelium -- abnormalitiesMutationRandomized Controlled Trials as TopicTreatment OutcomeVisual AcuityHumansUnited States
2. Human gene therapy for retinal disorders Publication: Silver Spring, MD : Center for Biologics Evaluation and Research, January 2020 Subject(s): Clinical Trials as TopicGenetic TherapyDrug Evaluation, PreclinicalRetinal Diseases -- therapyInvestigational New Drug ApplicationUnited StatesUnited States. Department of Health and Human Services.United States. Food and Drug Administration.
