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Neurology. 2003 May 27;60(10):1679-81.

Idebenone treatment in Friedreich's ataxia: neurological, cardiac, and biochemical monitoring.

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  • 1Department of Pediatric Neurology, University Hospitals Leuven, Belgium.


The authors report 1-year prospective data on eight patients with Friedreich ataxia. Idebenone did not halt the progression of ataxia. At the end of therapy, cardiac ultrasound demonstrated significant reduction of cardiac hypertrophy in six of eight patients. Cardiac strain and strain rate imaging showed that the reduction of hypertrophy is preceded by an early and linear improvement in cardiac function. Idebenone reduced erythrocyte protoporphyrin IX levels in five of six patients with elevated baseline levels; however, changes did not consistently relate to cardiac improvement.

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