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Idebenone in Friedreich's ataxia.

AbstractBACKGROUND:
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.
AuthorsCaterina Tonon, Raffaele Lodi
JournalExpert opinion on pharmacotherapy (Expert Opin Pharmacother) Vol. 9 Issue 13 Pg. 2327-37 (Sep 2008) ISSN: 1744-7666 [Electronic] England
PMID18710357 (Publication Type: Journal Article, Review)
Chemical References
  • Antioxidants
  • Ubiquinone
  • idebenone
Topics
  • Antioxidants (pharmacology, therapeutic use)
  • Clinical Trials as Topic
  • Dose-Response Relationship, Drug
  • Friedreich Ataxia (diagnosis, drug therapy, physiopathology)
  • Humans
  • Magnetic Resonance Angiography
  • Ubiquinone (analogs & derivatives, pharmacology, therapeutic use)

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