Duchenne muscular dystrophy future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Fahimeh Shojaei, M.D.

Future or Investigational Therapies

Future or investigational therapies for Duchenne muscular dystrophy include:

• Gene therapy
  • Injecting microdystrophin or minidystrophin genes through recombinant adeno-associated viral (rAAV) vectors
• Creatine monohydrate
  • Increases muscle strength
• Myostatin inactivation
  • Increase muscle mass and strength
• Cell therapy
  • Isolating and transplanting muscle satellite cells as a skeletal muscle progenitor.
• Idebenone (antioxidant)