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Drugs
Erschienen in: Drugs 7/2016

01.05.2016 | Adis Drug Evaluation

Idebenone: A Review in Leber’s Hereditary Optic Neuropathy

verfasst von: Katherine A. Lyseng-Williamson

Erschienen in: Drugs | Ausgabe 7/2016

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Abstract

Idebenone (Raxone®), a short-chain benzoquinone, is the only disease-specific drug approved to treat visual impairment in adolescents and adults with Leber’s hereditary optic neuropathy (LHON), a rare genetic mitochondrial disease that causes rapid and progressive bilateral vision loss. The mechanism of action of idebenone involves its antioxidant properties and ability to act as a mitochondrial electron carrier. Idebenone overcomes mitochondrial complex I respiratory chain deficiency in patients with LHON by transferring electrons directly to mitochondrial complex III (by-passing complex I), thereby restoring cellular energy (ATP) production and re-activating inactive-but-viable retinal ganglion cells, which ultimately prevents further vision loss and promotes vision recovery. The approval of idebenone in the treatment of LHON was based on the overall data from a randomized clinical trial, a follow-up study and real-world data. Taken together, these studies provide convincing evidence that oral idebenone 900 mg/day for 24 weeks has persistent beneficial effects in preventing further vision impairment and promoting vision recovery in patients with LHON relative to the natural course of the disease. Therefore, idebenone is a valuable agent to treat visual impairment in adolescents and adults with LHON.
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Metadaten
Titel
Idebenone: A Review in Leber’s Hereditary Optic Neuropathy
verfasst von
Katherine A. Lyseng-Williamson
Publikationsdatum
01.05.2016
Verlag
Springer International Publishing
Erschienen in
Drugs / Ausgabe 7/2016
Print ISSN: 0012-6667
Elektronische ISSN: 1179-1950
DOI
https://doi.org/10.1007/s40265-016-0574-3

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