The role of corticosteroids in muscular dystrophy: A critical appraisal

Corrado Angelini

Research output: Contribution to journalArticle

Abstract

Over the years various steroid trials have been conducted in Duchenne muscular dystrophy (DMD). In children who are still able to walk as well as in those who are wheelchair-bound, corticosteroids have been found to stabilize muscle strength for a period of time. Controlled clinical observations have shown that some boys remain ambulatory for years longer than reported in natural history data. The two main steroids used are prednisone/prednisolone and deflazacort. They are probably equally effective in stabilizing muscle strength but may have different side-effect profiles; for instance, deflazacort causes less weight gain. The exact mechanism by which steroids slow the dystrophic process is under investigation. DMD children treated long term also seem to develop other complications of the condition less frequently. For instance, they develop respiratory insufficiency later and have fewer cardiac symptoms. The therapeutic value of corticosteroids is limited, but these drugs represent the best treatment option currently available.

Original languageEnglish
Pages (from-to)424-435
Number of pages12
JournalMuscle and Nerve
Volume36
Issue number4
DOIs
Publication statusPublished - Oct 2007

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Keywords

  • Deflazacort
  • Duchenne muscular dystrophy
  • Functional outcome
  • Prednisolone
  • Prednisone
  • Respiratory insufficiency
  • Steroids
  • Trials
  • Weakness

ASJC Scopus subject areas

  • Clinical Neurology
  • Neuroscience(all)

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