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pubmed-article:15480852pubmed:abstractTextImpaired oxidative phosphorylation is a crucial factor in the pathogenesis of Friedreich's ataxia (FA). L-carnitine and creatine are natural compounds that can enhance cellular energy transduction. We performed a placebo-controlled triple-phase crossover trial of L-carnitine (3 g/d) and creatine (6.75 g/d) in 16 patients with genetically confirmed FA. Primary outcome measures were mitochondrial ATP production measured as phosphocreatine recovery by 31Phosphorus magnetic resonance spectroscopy, neurological deficits assessed by the international co-operative ataxia rating scale and cardiac hypertrophy in echocardiography. After 4 months on L-carnitine phosphocreatine recovery was improved compared to baseline (p<0.03, t-test) but comparison to placebo and creatine effects did not reach significance (p=0.06, F-test). Ataxia rating scale and echocardiographic parameters remained unchanged. Creatine had no effect in FA patients. L-carnitine is a promising substance for the treatment of FA patients, and larger trials are warranted.lld:pubmed
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pubmed-article:15480852pubmed:articleTitleL-carnitine and creatine in Friedreich's ataxia. A randomized, placebo-controlled crossover trial.lld:pubmed
pubmed-article:15480852pubmed:affiliationCentre of Neurology and Hertie-Institute for Clinical Brain Research, University of Tübingen, Germany. ludger.schoels@uni-tuebingen.delld:pubmed
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