Retinal Disease
ICER developed a report assessing the comparative clinical effectiveness and value of voretigene neparvovec (Luxturna®, Spark Therapeutics), a potentially one-time gene therapy for vision loss associated with biallelic RPE65-mediated retinal disease. Voretigene neparvovec is the first gene therapy to be approved by the US Food and Drug Administration (FDA). The appraisal committee vote revealed differences […]