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Article

The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil.

Ophthalmic genetics - 1 Aug 2022

Ferraz Sallum Juliana M, Godoy Juliana, Kondo Andrea, Kutner Jose Mauro, Vasconcelos Huber, Maia Andre

Abstract excerpt

PURPOSE: To report the first Brazilian patient with RPE65 deficiency-inherited retinal dystrophy (RPE65-IRD) treated with voretigene neparvovec-rzyl (VN). METHODS: An adult patient with Leber congenital amaurosis-2 with a homozygous mutation in the RPE65 gene (p.Phe83Leu) was treated bilaterally with VN. The clinical and surgical aspects are described. The baseline and 4-month postoperative ophthalmologic...

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Source

Identifiers

PMID: 35416119
DOI: 10.1080/13816810.2022.2053995

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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