Symptomatic and disease-modifying therapy for the progressive ataxias

Neurologist. 2004 Sep;10(5):275-89. doi: 10.1097/01.nrl.0000141651.35193.67.


Background: The progressive ataxias are a diverse group of neurologic diseases that share features of degeneration of the cerebellum and its inflow/outflow pathways but differ in etiology, course, and associated noncerebellar system involvement. Some will have treatable causes, but for most, the pathophysiology is incompletely known.

Review summary: Treatment strategies will include (1) definitive therapy when available, (2) symptomatic treatment and prevention of complications, and (3) rehabilitation and support resources. The physician will have to decide whether to introduce or approve the use of therapies based on as yet-unproven mechanisms or the use of complementary medicine approaches.

Conclusions: There are as yet no drugs that have been approved by the Food and Drug Administration for the treatment of the progressive ataxias and relatively few disease-modifying therapies, but symptomatic and rehabilitation interventions can greatly improve the quality of life of individuals with these disabling neurodegenerative disorders.

Publication types

  • Review

MeSH terms

  • Ataxia / diagnosis
  • Ataxia / pathology
  • Ataxia / physiopathology*
  • Ataxia / therapy*
  • Cell Cycle / physiology
  • Cerebellum / pathology
  • Cerebellum / physiopathology*
  • Complementary Therapies
  • Humans
  • Ion Channels / physiology
  • Metabolism, Inborn Errors / genetics
  • Metabolism, Inborn Errors / physiopathology
  • Metabolism, Inborn Errors / therapy
  • Mitochondria / metabolism
  • Nervous System Diseases / diagnosis
  • Nervous System Diseases / pathology
  • Nervous System Diseases / physiopathology
  • Nervous System Diseases / therapy
  • Trinucleotide Repeat Expansion


  • Ion Channels