Botulinum toxin in clinical practice

J Neurol Neurosurg Psychiatry. 2004 Jul;75(7):951-7. doi: 10.1136/jnnp.2003.034702.


Botulinum toxin, the most potent biological toxin, has become a powerful therapeutic tool for a growing number of clinical applications. This review draws attention to new findings about the mechanism of action of botulinum toxin and briefly reviews some of its most frequent uses, focusing on evidence based data. Double blind, placebo controlled studies, as well as open label clinical trials, provide evidence that, when appropriate targets and doses are selected, botulinum toxin temporarily ameliorates disorders associated with excessive muscle contraction or autonomic dysfunction. When injected not more often than every three months, the risk of blocking antibodies is slight. Long term experience with this agent suggests that it is an effective and safe treatment not only for approved indications but also for an increasing number of off-label indications.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Autonomic Nervous System Diseases / drug therapy*
  • Botulinum Toxins, Type A* / immunology
  • Botulinum Toxins, Type A* / pharmacology
  • Botulinum Toxins, Type A* / therapeutic use
  • Dystonia / drug therapy*
  • Gastrointestinal Diseases / drug therapy*
  • Gastrointestinal Motility / drug effects
  • Humans
  • Membrane Proteins / metabolism
  • Movement Disorders / drug therapy
  • Nerve Tissue Proteins / metabolism
  • Neuromuscular Agents* / immunology
  • Neuromuscular Agents* / pharmacology
  • Neuromuscular Agents* / therapeutic use
  • Presynaptic Terminals / drug effects
  • Qa-SNARE Proteins
  • R-SNARE Proteins
  • Spasm / drug therapy*
  • Synaptosomal-Associated Protein 25


  • Membrane Proteins
  • Nerve Tissue Proteins
  • Neuromuscular Agents
  • Qa-SNARE Proteins
  • R-SNARE Proteins
  • SNAP25 protein, human
  • Synaptosomal-Associated Protein 25
  • Botulinum Toxins, Type A