Idebenone in Friedreich's ataxia

Expert Opin Pharmacother. 2008 Sep;9(13):2327-37. doi: 10.1517/14656566.9.13.2327.


Background: 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.

Objective: The aim of this study was to evaluate the efficacy of idebenone administration and define the optimal dosage.

Methods: A critical evaluation of all open and double-blinded idebenone trials in Friedreich's ataxia patients was undertaken.

Results/conclusions: 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.

Publication types

  • Review

MeSH terms

  • Antioxidants / pharmacology
  • Antioxidants / therapeutic use*
  • Clinical Trials as Topic
  • Dose-Response Relationship, Drug
  • Friedreich Ataxia / diagnosis
  • Friedreich Ataxia / drug therapy*
  • Friedreich Ataxia / physiopathology
  • Humans
  • Magnetic Resonance Angiography
  • Ubiquinone / analogs & derivatives*
  • Ubiquinone / pharmacology
  • Ubiquinone / therapeutic use


  • Antioxidants
  • Ubiquinone
  • idebenone