Efficacy and Safety of Voretigene Neparvovec in RPE65 -Retinopathy: Results of a Phase III Trial in Japan.
Fujinami Kaoru, Akiyama Kunihiko, Tsunoda Kazushige, Ito Saori, Seko Noriko, Yamamoto Shuichi
Voretigene neparvovec gene therapy improved light sensitivity and visual fields in Japanese RPE65-retinopathy patients over one year, with a good safety profile, supporting its broader clinical use.