Interim Results From a Multicenter Observational Safety Study of Patients Treated With Voretigene Neparvovec-rzyl in the United States.

With a median 3.7 years of follow-up (range, 2.9-4.9), most patients (95%) reported ≥1 treatment-emergent adverse events (387 events); 67% were procedure related, 9% were attributed to VN, and 82% were mild.

The area of chorioretinal atrophy mostly localized to the retinotomy or bleb area, with 40% of eyes with chorioretinal atrophy having atrophy outside the bleb area and highly correlated between fellow eyes (r=0.97; P<0.0001).