Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy. Journal Abstract

Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy.

Publication Date: 2024 Dec 19

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Authors

Patrick Yu-Wai-Man; Nancy J Newman; Valérie Biousse; Valerio Carelli; Mark L Moster; Catherine Vignal-Clermont; Thomas Klopstock; Alfredo A Sadun; Robert C Sergott; Rabih Hage; Simona Degli Esposti; Chiara La Morgia; Claudia Priglinger; Rustum Karanja; Magali Taiel; José-Alain Sahel

Abstract

OBJECTIVE

Limited studies have assessed the long-term benefit/risk of gene therapy for Leber hereditary optic neuropathy (LHON).

Source

JAMA ophthalmology

Pub Types(s)

Journal Article

Language

English

PubMed ID

39699886