Structural, functional and patient-reported 24-month outcomes of voretigene neparvovec in Portuguese patients with RPE65-associated disease
Eye (Lond). 2026 Apr 17. doi: 10.1038/s41433-026-04467-4. Online ahead of print. ABSTRACT BACKGROUND/OBJECTIVES: Voretigene neparvovec (VN) is a one-time gene therapy for biallelic RPE65-associated retinal degeneration…