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Real-World Evidence: How Idebenone Helps Restore Vision in Leber’s Hereditary Optic Neuropathy

Leber’s Hereditary Optic Neuropathy (LHON) is a genetic mitochondrial disorder that causes rapid, irreversible vision loss. Because LHON originates from Complex I dysfunction in the mitochondrial respiratory chain, conventional treatments offer limited benefit.
Idebenone, however, bypasses the defective complex and restores electron flow, helping retinal ganglion cells recover function. Studies published in the Journal of Neuro-Ophthalmology confirm that patients using Idebenone recover and maintain more visual function than untreated groups. Real-world data shows the best outcomes occur when treatment begins early and continues for at least 24 months.
This vision recovery is not temporary — it reflects improved mitochondrial energy availability, reduced oxidative stress, and enhanced cell survival. Idebenone remains the only clinically approved treatment for LHON in the UK and EU and is considered the frontline therapy worldwide.

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