Pharmacological treatments for Friedreich ataxia ( Cochrane Database of Systematic Reviews 2026)

Lyons S, Kearney M, Fahey MC, Janjal P, Pandolfo M, Patton P. Pharmacological treatments for Friedreich ataxia. Cochrane Database of Systematic Reviews 2026, Issue 5. Art. No.: CD007791. DOI: 10.1002/14651858.CD007791.pub5. Accessed 12 May 2026.

In this updated Cochrane systematic review, meta‐analysis of results on the ataxia rating scale showed that pharmacological treatments probably make little or no difference compared with placebo after 12 months of treatment. Given this result, the probable improvement that we found in upper limb dexterity was unexpected. Treatment‐emergent adverse events leading to cessation of medication or death may be no more common in treatment groups than placebo groups as there were few adverse events detected in the treated groups. However, the studies may not have detected all rare and serious adverse events.

Pharmacological treatments for Friedreich ataxia