2024
DOI: 10.1001/jamaophthalmol.2024.5375
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Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy

Patrick Yu-Wai-Man,
Nancy J. Newman,
Valérie Biousse
et al.

Abstract: ImportanceLimited studies have assessed the long-term benefit/risk of gene therapy for Leber hereditary optic neuropathy (LHON).ObjectiveTo determine the safety and efficacy of lenadogene nolparvovec in patients with LHON due to the MT-ND4 gene variant for up to 5 years after administration.Design, Setting, and ParticipantsThe RESCUE and REVERSE Long-Term Follow-up Study (RESTORE), conducted from 2018 to 2022, is the 5-year follow-up study of the 2 phase 3 clinical studies RESCUE (Efficacy Study of Lenadogene … Show more

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