Friedreich ataxia (FRDA) is the most common autosomal recessive
ataxia. Oxidative damage within the mitochondria seems to have a key role in the disease phenotype. Therefore, FRDA treatment options have been mostly directed at
antioxidant protection against mitochondrial damage. Available evidence seems to suggest that patients with FRDA should be treated with
idebenone, because it is well tolerated and may reduce
cardiac hypertrophy and, at higher doses, also improve neurological function, but large controlled clinical trials are still needed. Alternatively, gene-based strategies for the treatment of FRDA may involve the development of small-molecules increasing
frataxin gene transcription. Animal and human studies are strongly needed to assess whether any of the potential new treatment strategies, such as
iron-chelating
therapies or treatment with
erythropoietin or
histone deacetylase inhibitors and other gene-based strategies, may translate into an effective
therapy for this devastating disorder. In this review, we try to provide an answer to some questions related to current and emerging treatment options in the management of FRDA.