Which medications are used in the treatment of Duchenne muscular dystrophy (DMD)?

Updated: Aug 17, 2020
  • Author: Twee T Do, MD; Chief Editor: Jeffrey D Thomson, MD  more...
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Answer

Several agents have shown promise in management of Duchenne muscular dystrophy (MD). The mainstay of therapy has been the steroids; however, the lack of prolonged efficacy and the attendant adverse effects of higher-dose steroids limit their use. Deflazacort, an oxazoline derivative of prednisolone, may offer therapeutic efficacy with fewer adverse effects. The approval of eteplirsen, the first phosphorodiamidate morphino oligomer (PMO), provides an addition to the therapeutic armamentarium; however, continued approval of eteplirsen is contingent on postmarketing performance data.


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