Expert Opinion on Pharmacotherapy 2008-09-01

Idebenone in Friedreich's ataxia.

Caterina Tonon, Raffaele Lodi

文献索引：Expert Opin. Pharmacother. 9(13) , 2327-37, (2008)

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摘要

Friedreich's ataxia is an autosomal recessive neurodegenerative disease where impaired mitochondrial function and excessive production of free radicals play a central pathogenetic role. Idebenone, a synthetic analogue of coenzyme Q, is a powerful antioxidant that was first administrated to Friedreich's ataxia patients less than 10 years ago.The aim of this study was to evaluate the efficacy of idebenone administration and define the optimal dosage.A critical evaluation of all open and double-blinded idebenone trials in Friedreich's ataxia patients was undertaken.Idebenone is well tolerated in paediatric and adult patients. Most trials demonstrated a positive effect on cardiac hypertrophy. The neurological function is in general not modified in adult patients, but a dose-dependent effect was demonstrated in young Friedreich's ataxia patients. Further double-blinded high-dose trials should evaluate idebenone in Friedreich's ataxia early in the disease course.