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
