Approaching a New Age in Duchenne Muscular Dystrophy Treatment

Research output: Contribution to journalArticlepeer-review

Abstract

Duchenne muscular dystrophy is the most common and severe form of muscular dystrophy. The cornerstones of current treatment include corticosteroids for skeletal muscle weakness, afterload reduction for cardiomyopathy, and noninvasive ventilation for respiratory failure. With these interventions, patients are walking and living longer. However, the current status is still far from adequate. Increased private and federal funding of studies in Duchenne muscular dystrophy has led to a large number of novel agents with propitious therapeutic potential. These include agents that modify dystrophin expression, increase muscle growth and regeneration, and modulate inflammatory responses. Many of these agents are already in clinical trials. Challenges to the development of additional novel therapeutics exist, including lack of validated animal models and lack of adequate biomarkers as surrogate endpoints. However, these challenges are not insurmountable and the next decade will likely see meaningful, new treatment options introduced into the clinical care of patients with Duchenne muscular dystrophy.

Original languageEnglish (US)
Pages (from-to)583-591
Number of pages9
JournalNeurotherapeutics
Volume5
Issue number4
DOIs
StatePublished - Oct 2008

Keywords

  • Duchenne
  • clinical trials
  • gene therapy
  • muscular dystrophy
  • myostatin
  • therapeutics

ASJC Scopus subject areas

  • Pharmacology
  • Clinical Neurology
  • Pharmacology (medical)

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