Leber hereditary optic neuropathy (LHON) is a mitochondrial disorder causing acute or subacute visual failure. Consensus guidelines identify a specific time window from symptom onset during which pharmacological intervention is indicated.
This protocol applies to LHON patients presenting with vision loss of less than one year in duration — the window in which treatment is guideline-supported. Timing from onset is a key eligibility criterion for the recommended approach.
Guidelines support an orally administered agent taken on a daily basis over a defined treatment course. The complete regimen, eligibility details, and monitoring parameters are contained in the full protocol.
Idebenone, the only medication approved by the European Medicine Agency to treat LHON, is a synthetic form of coenzyme Q10, a molecule that acts as an electron shuttle in the electron transport chain during mitochondrial respiration.
In 2011, a prospective, randomized clinical trial (RHODOS) randomized patients with LHON diagnosed within the previous five years to treatment with 24 weeks of daily oral idebenone or placebo.
Consensus guidelines suggest starting idebenone for LHON patients with vision loss of less than one year in duration.
View source ↗