The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil.
Juliana Maria Ferraz SallumJuliana Aparecida Preto de GodoyAndrea Tiemi KondoJosé Mauro KutnerHuber Martins VasconcelosAndré MaiaPublished in: Ophthalmic genetics (2022)
This case reinforces the clinical trial results and proves that the procedure is feasible in countries such as Brazil.