Antioxidant Treatment of Patients With Friedreich Ataxia

• DOI: 10.1001/archneur.62.4.621
• Publication Date: 2005-04-11T17:25:50Z
• Authors: Paul E. Hart; Raffaele Lodi; Bheeshma Rajagopalan; Jane L. Bradley; Jenifer G. Crilley; Christopher Turner; Andrew M. Blamire; David Manners; Peter Styles; Anthony H. V. Sch...; J. Mark Cooper
• Keywords: Bioenergetics; Coenzyme Q10

Decreased mitochondrial respiratory chain function and increased oxidative stress have been implicated in the pathogenesis of Friedreich ataxia (FRDA), raising possibility that energy enhancement antioxidant therapies may be an effective treatment.To evaluate long-term efficacy a combined therapy on bioenergetics clinical course FRDA.Open-labeled pilot trial over 47 months.Patients Seventy-seven patients with genetically defined FRDA. Intervention A coenzyme Q(10) (400 mg/d) vitamin E (2100 IU/d) 10 FRDA months.Clinical assessment using echocardiography International Cooperative Ataxia Rating Scale cardiac skeletal muscle as assessed phosphorus P 31 magnetic resonance spectroscopy.There was significant improvement maintained throughout months therapy. Echocardiographic data revealed significantly fractional shortening at 35- 47-month time points. Comparison cross-sectional from 77 indicated changes total kinetic scores period were better than predicted for 7 patients, but posture gait hand dexterity progressed predicted.This resulted sustained synthesis associated slowing progression certain features function.