Efficacy and safety of voretigene neparvovec in RPE65-retinopathy: Results of a phase 3 trial in Japan
We report the efficacy and safety of voretigene neparvovec (VN) as an adeno-associated viral vector-based gene therapy for Japanese patients with inherited retinal dystrophy (IRD) caused by biallelic pathogenic RPE65 variants (RPE65-retinopathy).