Initial findings from the real-world PERCEIVE study of voretigene neparvovec in patients with RPE65 mutation-associated inherited retinal dystrophy show safety and efficacy in line with those observed in pivotal trials.
“Despite limited availability of data at the time of this first analysis, the safety and tolerability of [voretigene neparvovec] observed in patients are consistent with its known safety profile,” M. Dominik Fischer, MD, said at the virtual Euretina congress.
Inherited retinal dystrophy caused by biallelic RPE65 mutation is a rare genetic condition that can