Journal
EXPERT OPINION ON PHARMACOTHERAPY
Volume 9, Issue 13, Pages 2327-2337Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1517/14656566.9.13.2327
Keywords
antioxidant; end-points; Friedreich's ataxia; idebenone
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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.
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