Duchenne Muscular Dystrophy (DMD)

The Disease:

  • Primarily affects boys
  • Symptoms include progressive muscle weakness and atrophy in early childhood, which rapidly results in loss of ambulation and respiratory muscle function
  • Often causes cardiomyopathy in adolescence and death typically occurs in early adulthood
  • Approximately one in every 3,500 boys in U.S. is born with DMD and approximately 15,000 people in the U.S. are currently living with DMD
  • No approved disease-modifying therapies
  • Current standard of care consists of palliative measures

The Editas Approach

We believe that restoring dystrophin activity before the onset of severe loss of muscle function could significantly and favorably alter disease progression. Our genome editing approach is to introduce targeted deletions of mutation-containing segments of the gene in order to create smaller, yet functional versions of the dystrophin gene.