Raxone (idebenone) in LHON - An update

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Abstract

Leber’s Hereditary Optic Neuropathy (LHON) is caused by mtDNA mutations affecting subunits of Complex I of the mitochondrial electron transport chain. Affected mutation carriers suffer from rapidly progressing and mostly irreversible bilateral vision loss. An increasing body of evidence indicates that idebenone has therapeutic potential for the treatment of LHON. Data from a randomized placebo-controlled study (RHODOS) and from a number of case reports and retrospective cohort studies demonstrate that patients with established vision loss may benefit from idebenone treatment and recover visual acuity. Early treatment start (e.g. within one year from onset of symptoms) appears to be associated with better outcome, which is supported by the cellular pathology of LHON. Data from a currently on-going named patient program (NPP), where LHON patients receive 900 mg/day Raxone® under routine clinical care, will be presented and an update on the regulatory status of Raxone® provided.

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