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Clinical trials in Leber hereditary optic neuropathy: outcomes and opportunities.

Current opinion in neurology - 1 Feb 2025

Chen Benson S, Newman Nancy J

Abstract excerpt

PURPOSE OF REVIEW: Leber hereditary optic neuropathy (LHON) is a mitochondrial DNA disease characterised by sequential bilateral vision loss due to loss of retinal ganglion cells. The purpose of this review is to provide an update on the results of recent clinical trials for LHON, focusing on studies of idebenone and lenadogene nolparvovec gene therapy. RECENT FINDINGS: Evidence from three clinical studies...

Topics

Optic Atrophy, Hereditary, Leber
Humans
Genetic Therapy
Ubiquinone
Clinical Trials as Topic
Treatment Outcome
DNA, Mitochondrial
Antioxidants
Mutation

Source

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Identifiers

PMID: 39704163
DOI: 10.1097/WCO.0000000000001343

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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