Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial

• Published in: Lancet neurology Vol. 6; no. 10; pp. 878 - 886
• Main Authors: Di Prospero, Nicholas A, MD, Baker, Angela, BSN, Jeffries, Neal, PhD, Fischbeck, Kenneth H, MD
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.10.2007; Elsevier Limited
• Subjects: Activities of Daily Living; Adolescent; Antioxidants - administration & dosage; Antioxidants - adverse effects; Antioxidants - therapeutic use; Ataxia; Benzoquinones - administration & dosage; Benzoquinones - adverse effects; Benzoquinones - therapeutic use; Child; Deoxyguanosine - analogs & derivatives; Deoxyguanosine - urine; DNA Damage; Dose-Response Relationship, Drug; Drug dosages; Female; Friedreich Ataxia - drug therapy; Friedreich Ataxia - genetics; Friedreich Ataxia - physiopathology; Friedreich Ataxia - urine; Humans; Male; Nervous System - drug effects; Nervous System - physiopathology; Neurology; Neutropenia - chemically induced; Severity of Illness Index; Testing laboratories; Ubiquinone - analogs & derivatives; United States > US; Switzerland

Summary Background Friedreich's ataxia (FA) is a progressive, multisystem, degenerative disorder caused by a reduction in frataxin. Loss of frataxin results in mitochondrial dysfunction and oxidative damage in patients and model systems. Previous studies have indicated that the antioxidant ideb...