welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

key information

study id #: NCT02420379

condition: Duchenne Muscular Dystrophy (DMD)

status: completed


This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

intervention: eteplirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT02420379

last updated: August 01, 2020