1.
Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis.
Deng Callie, Zhao Peter Y, Branham Kari, Schlegel Dana, Fahim Abigail T, Jayasundera Thiran K et al.
Graefes Arch Clin Exp OphthalmolJan 20220 citationsRetrospective Studies
Real-world data on voretigene neparvovec for pediatric RPE65-LCA shows significant, sustained visual function improvements, confirming its safety and efficacy for clinical use.