Symptomatic and disease-modifying therapy for the progressive ataxias.
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.


RESULTS
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.
Perlman
Susan L
SL
David Geffen School of Medicine at the University of California, Los Angeles 90095, USA. sperlman@ucla.edu
D001259:Ataxia / Q000175:diagnosis / Q000473:pathology / Q000503:physiopathology* / Q000628:therapy*; D002453:Cell Cycle / Q000502:physiology; D002531:Cerebellum / Q000473:pathology / Q000503:physiopathology*; D000529:Complementary Therapies; D006801:Humans; D007473:Ion Channels / Q000502:physiology; D008661:Metabolism, Inborn Errors / Q000235:genetics / Q000503:physiopathology / Q000628:therapy; D008928:Mitochondria / Q000378:metabolism; D009422:Nervous System Diseases / Q000175:diagnosis / Q000473:pathology / Q000503:physiopathology / Q000628:therapy; D019680:Trinucleotide Repeat Expansion
D007473:Ion Channels