Idebenone in Friedreich's ataxia.
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.


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.
Tonon
Caterina
C
Università di Bologna, Dipartimento di Medicina Interna, dell'Invecchiamento e Malattie Nefrologiche, Azienda Ospedaliero-Universitaria di Bologna, Via Massarenti 9, 40138 Bologna, Italy.
Lodi
Raffaele
R
D000975:Antioxidants / Q000494:pharmacology / Q000627:therapeutic use*; D002986:Clinical Trials as Topic; D004305:Dose-Response Relationship, Drug; D005621:Friedreich Ataxia / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D018810:Magnetic Resonance Angiography; D014451:Ubiquinone / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use
D000975:Antioxidants; D014451:Ubiquinone; C036619:idebenone