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Article

A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy.

Brain : a journal of neurology - 1 Sept 2011

Klopstock Thomas, Yu-Wai-Man Patrick, Dimitriadis Konstantinos, Rouleau Jacinthe, Heck Suzette, Bailie Maura, Atawan Alaa, Chattopadhyay Sandip, Schubert Marion, Garip Aylin, Kernt Marcus, Petraki Diana, Rummey Christian, Leinonen Mika, Metz Günther, Griffiths Philip G, Meier Thomas, Chinnery Patrick F

Abstract excerpt

Major advances in understanding the pathogenesis of inherited metabolic disease caused by mitochondrial DNA mutations have yet to translate into treatments of proven efficacy. Leber's hereditary optic neuropathy is the most common mitochondrial DNA disorder causing irreversible blindness in young adult life. Anecdotal reports support the use of idebenone in Leber's hereditary optic neuropathy, but this has not...

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Identifiers

PMID: 21788663
DOI: 10.1093/brain/awr170
PMCID: PMC3170530

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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