Caterina Tonon1, Raffaele Lodi. 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.
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 ataxiapatients 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 ataxiapatients 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 ataxiapatients. Further double-blinded high-dose trials should evaluate idebenone in Friedreich's ataxia early in the disease course.
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