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Ataxia Treatments: An Evidence Review

Information sourced from NEJM Journal Watch:

What Is the Evidence for Current Ataxia Treatments?

The American Academy of Neurology guideline subcommittee reviewed the evidence.

Sponsoring Organization: The guideline development, dissemination, and implementation subcommittee of the American Academy of Neurology

Target Population: Patients with cerebellar motor dysfunction and ataxia

Background and Objective

A subcommittee of the American Academy of Neurology reviewed the current evidence on the treatment of cerebellar motor dysfunction and ataxia.

Key Points

  • For episodic ataxia type 2, 4-aminopyridine (15 mg/day) “probably reduces” frequency of ataxia attacks (based on 1 Class I study lasting 3 months).

  • For ataxia of mixed etiology, riluzole “probably improves” ataxia signs (1 Class I study lasting 8 weeks).

  • For Friedreich ataxia or spinocerebellar ataxia (SCA), riluzole “probably improves” ataxia signs (1 Class I study lasting 12 months).

  • For SCA type 3, high-dose valproic acid (1200 mg/day) “possibly improves” ataxia (1 class II study lasting 12 weeks).

  • For spinocerebellar degeneration, thyrotropin-releasing hormone “possibly improves” some ataxia signs (1 Class II study lasting 2 weeks).

  • For Friedreich ataxia, deferiprone “possibly worsens” ataxia signs (1 Class II study lasting 6 months).

  • For degenerative ataxias, an inpatient rehabilitation program “probably improves” ataxia and function (1 Class I study lasting 4 weeks), and transcranial magnetic stimulation “possibly improves” cerebellar motor signs (1 Class II study lasting 21 days).

  • Too few results are available to recommend for or against use of stochastic whole-body vibration therapy (1 Class III study).


This guideline is useful, as many clinicians may have assumed that there are no treatments for cerebellar motor dysfunction and ataxia. Clinicians should pay attention to the type of ataxia and remember that 4-aminopyridine, riluzole, valproic acid, transcranial magnetic stimulation, and rehabilitation all may improve some ataxia symptoms, but improvement generally depends on the ataxia subtype and method of applying the therapy. Lithium and deferiprone may worsen some ataxias. Although the effects of these therapies may be mild, improvement could be meaningful for individual patients with ataxia.

Michael S. Okun, MD reviewing Zesiewicz TA et al. Neurology 2018 Mar.


Zesiewicz TA et al. Comprehensive systematic review summary: Treatment of cerebellar motor dysfunction and ataxia: Report of the Guideline Development, Dissemination, and Implementation Subcommittee of the American Academy of Neurology. Neurology 2018 Mar; 90:464.

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