Idebenone in Friedreich's ataxia

  title={Idebenone in Friedreich's ataxia},
  author={Caterina Tonon and Raffaele Lodi},
  journal={Expert Opinion on Pharmacotherapy},
  pages={2327 - 2337}
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… 

New developments in pharmacotherapy for Friedreich ataxia

In this review article, the authors discuss the current and prior in vivo and in vitro research studies related to the treatment of FRDA, with a particular interest in future implications of each therapy.

Neuro-Ophthalmological Findings in Friedreich’s Ataxia

This review provides a brief overview of the main aspects of FRDA and then focuses on the ocular involvement of this pathology and the possible use of retinal biomarkers.

Intermediate-dose idebenone and quality of life in Friedreich ataxia.

Ocular Involvement in Friedreich Ataxia Patients and Its Relationship with Neurological Disability, a Follow-Up Study

The VF and the OCT could be useful biomarkers in Friedreich ataxia, both for their correlation with neurological disease as well as for their ability to evaluate disease progression.

A Strategy for Suppressing Redox Stress within Mitochondria.

The aza analogue of the experimental neuroprotective drug idebenone quenches lipid peroxidation more effectively than α-tocopherol and potently suppresses reactive oxygen species in cells under oxidative stress via a catalytic cycle in which both forms of oxidative stress are suppressed simultaneously.

Electron transfer mediators and other metabolites and cofactors in the treatment of mitochondrial dysfunction.

The rationale for the use of ETC cofactors, other metabolites secondarily decreased in MDs, antioxidants, and agents acting on lactic acidosis are reviewed.



Idebenone and reduced cardiac hypertrophy in Friedreich's ataxia

There is a good case for giving idebenone continuously in a dose of 5–10 mg/kg/day in patients with Friedreich's ataxia at the onset of hypertrophic cardiomyopathy, as the drug has no serious side effects.

Heart Hypertrophy and Function Are Improved by Idebenone in Friedreich's Ataxia

An open trial of idebenone (oral supplementation; 5 mg/kg/day) in a large series of FRDA patients and followed their left ventricular mass and function, demonstrating the efficiency of Idebenone in controlling heart hypertrophy in FRDA.

Friedreich's ataxia: idebenone treatment in early stage patients.

Idebenone treatment at early stages of the disease seems to reduce the progression of cerebellar manifestations and further blind trials with a greater number of patients and higher doses are needed to fully assess the therapeutic potential of idebenone in Friedreich's ataxia.

Idebenone in patients with Friedreich ataxia

Idebenone treatment in paediatric and adult patients with Friedreich ataxia: long-term follow-up.

  • M. PinedaJ. Arpa R. Artuch
  • Medicine
    European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society
  • 2008

Friedreich's ataxia.

Antioxidant enzymes in blood of patients with Friedreich's ataxia

Data show an impairment in vivo of antioxidant enzymes in patients with Friedreich's ataxia and provide evidence of an increased sensitivity to oxidative stress, supporting a consistent role of free radical cytotoxicity in the pathophysiology of the disease.

Antioxidant treatment of patients with Friedreich ataxia: four-year follow-up.

This therapy resulted in sustained improvement in mitochondrial energy synthesis that was associated with a slowing of the progression of certain clinical features and a significant improvement in cardiac function.