Drugs in selected ataxias

Abstract

Options for drug treatment are very limited in patients with degenerative forms of ataxias. Drug treatments are available in a few metabolic disorders and vitamin defi ciencies that can cause ataxia. These diseases have to be excluded in all cases of ataxias of unknown origin. Despite the huge increase in knowledge in the underlying genetics and pathophysiology, however, causal treatments are currently not available for the large majority of hereditary and non-hereditary degenerative ataxias. Most preclinical and clinical studies have been done in Friedreich’s ataxia, and fi ndings will be briefl y summarized. Likewise, no symptomatic drug treatment is available which ameliorates the clinical signs and symptoms of ataxia. The only exceptions are aminopyridines for treatment of downbeat nystagmus, and acetazolamide and aminopyridines for treatment of episodic ataxias. A number of other drugs have been tried for symptomatic treatment of ataxias, but without provable effects.