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Drugs Today 2016, 52(3): 173
ISSN 1699-3993
Copyright 2016 Clarivate Analytics
CCC: 1699-3993
DOI: 10.1358/dot.2016.52.3.2463564
Idebenone for Leber's hereditary optic neuropathy
Gueven, N.
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[R]) 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.

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