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Idebenone for Leber's hereditary optic neuropathy


Gueven, N, Idebenone for Leber's hereditary optic neuropathy, Drugs of today, 52, (3) pp. 173-181. ISSN 1699-3993 (2016) [Refereed Article]

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Copyright 2016 Prous Science, S.A.U.

DOI: doi:10.1358/dot.2016.52.3.2463564


Idebenone is a rapidly absorbed, safe and well-tolerated drug and is currently the only clinically proven treatment option for Leber's hereditary optic neuropathy (LHON) patients. Idebenone (Raxone®) is approved by the European Medicines Agency for the treatment of LHON and has been available on the European market since 2015. Due to its molecular mode of action of bypassing the defective mitochondrial complex I, idebenone leads to improved energy supply and a functional recovery of retinal ganglion cells during the acute stage of the disease, thereby preventing further vision loss and promoting recovery of vision. Thus, commencing treatment shortly after the onset of symptoms is likely to have the best therapeutic effect, a hypothesis that is supported by the available clinical data.

Item Details

Item Type:Refereed Article
Keywords:genetic disorders, idebenone, inherited mitochondrial respiratory chain diseases, LHON, Leberís hereditary optic neuropathy, metabolic diseases
Research Division:Biomedical and Clinical Sciences
Research Group:Pharmacology and pharmaceutical sciences
Research Field:Clinical pharmacology and therapeutics
Objective Division:Expanding Knowledge
Objective Group:Expanding knowledge
Objective Field:Expanding knowledge in the biological sciences
UTAS Author:Gueven, N (Dr Nuri Guven)
ID Code:109158
Year Published:2016
Web of Science® Times Cited:18
Deposited By:Pharmacy
Deposited On:2016-05-30
Last Modified:2017-11-06

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