New therapies in muscular dystrophies

C. Angelini, D. M. Bonifati

Research output: Contribution to journalArticlepeer-review

Abstract

Muscular dystrophies are a group of hereditary muscle disorders that often result in severe disability. Curative therapy is not yet available for muscular dystrophies (MD). In the near future, it is not expected that gene-replacement therapy will be available. Other strategies to decrease the rate of muscle necrosis and to increase strength in patients are necessary. Therefore the interest in symptomatic drug treatment has recently increased. A few trials have been performed on different types of muscular dystrophies, and some have generated positive results on muscle strength or muscle mass. We review the state of the art in therapy of MD and summarize the drugs that have been used and the evidence and results of such clinical trials.

Original languageEnglish
JournalNeurological Sciences
Volume21
Issue number5 SUPPL.
Publication statusPublished - 2000

Keywords

  • Dystrophinopathies
  • Muscular dystrophies
  • Therapy

ASJC Scopus subject areas

  • Neuroscience(all)
  • Clinical Neurology
  • Psychiatry and Mental health
  • Dermatology

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