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Neurological eff ects of high-dose idebenone in patients with Friedreich ’ s ataxia : a randomised , placebo-controlled trial

Background Friedreich’s ataxia (FA) is a progressive, multisystem, degenerative disorder caused by a reduction in frataxin. Loss of frataxin results in mitochondrial dysfunction and oxidative damage in patients and model systems. Previous studies have indicated that the antioxidant idebenone (5 mg/kg daily) reduces cardiac hypertrophy, but defi nite improvement in neurological function has not been shown.