ROME — Voretigene neparvovec proved successful as a treatment for Leber congenital amaurosis 2 in preschool children, as shown by a study presented at the FLORetina-ICOOR meeting.
The study was carried out at the University of Munich in a group of five children with molecularly confirmed RPE65 mutation-associated Leber congenital amaurosis 2 (LCA2), the youngest to be treated with Luxturna (voretigene neparvovec, Novartis) in Germany so far.
“There are challenges in delivering this treatment in children,” Siegfried G. Priglinger, MD, PhD, said. The pars plana is different, the