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Leber hereditary optic neuropathy (LHON) causes irreversible blindness in young-adult life and is the most common mitochondrial DNA disorder. Idebenone is a potent antioxidant and inhibitor of lipid peroxidation, interacting with the mitochondrial electron transport chains; it is a synthetic analogue of coenzyme Q10. Because small, uncontrolled studies suggested a benefit in LHON, these researchers conducted a manufacturer-sponsored, 24-week, multicenter, double-blind, randomized, placebo-controlled trial of idebenone (900 mg/day) involving 85 patients with LHON. The primary end point was the best recovery of visual acuity between baseline and week 24.
The treatment and placebo groups did not differ in the primary endpoint; the change in log…