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

Idebenone in Friedreich's ataxia.

Author information

1
Università di Bologna, Dipartimento di Medicina Interna, dell'Invecchiamento e Malattie Nefrologiche, Azienda Ospedaliero-Universitaria di Bologna, Via Massarenti 9, 40138 Bologna, Italy.

Abstract

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.

PMID:
18710357
DOI:
10.1517/14656566.9.13.2327
[Indexed for MEDLINE]

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