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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
Ophthalmol SciJul 20250 citationsClinical Trial
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.