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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)
 

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Idebenone in patients with Friedreich ataxia.

Friedreich ataxia ( FA), the most common form of degenerative ataxia, is thought to be caused by respiratory deficiency due to mitochondrial iron accumulation and oxidative stress. Idebenone, a free-radical scavenger, protects mitochondrial function in in vitro models of FA. In a placebo-controlled crossover trial we studied the effect of idebenone on respiratory function in nine ambulant FA patients. (31)P magnetic resonance spectroscopy demonstrated mitochondrial impairment in vivo in skeletal muscle of all FA patients, but no recovery with idebenone. No effects were seen in clinical scores. Echocardiography did not confirm a preliminary study reporting improvement of FA- associated cardiomyopathy with idebenone.[1]

References

  1. Idebenone in patients with Friedreich ataxia. Schöls, L., Vorgerd, M., Schillings, M., Skipka, G., Zange, J. Neurosci. Lett. (2001) [Pubmed]
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