Duchenne muscular dystrophy (DMD) is a genetically inherited neuromuscular disease that almost entirely affects boys and results in a progressive loss of muscle function, resulting in progressive weakness and eventual death usually from cardiac and respiratory failure. It is the most common pediatric muscular dystrophy with a prevalence of one in 3,500 – 5,000 live male births, or about 400 to 600 boys per year in the US.

Treatments of Interest:

  • deflazacort (Emflaza®, PTC Therapeutics)
  • Two exon-skipping therapies: eteplirsen (Exondys 51®, Sarepta) and golodirsen (Sarepta).

Eteplirsen was approved by the FDA in September 2016 for patients with a confirmed mutation in the dystrophin gene that is amenable to exon 51 skipping. Sarepta has announced its intention to file for accelerated approval of golodirsen by the end of 2018.

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Final Documents

Below you will find the final documents from the assessment review process: