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completed
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
study id #: NCT02420379
condition: Duchenne Muscular Dystrophy (DMD)
status: completed
purpose: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: March 12, 2019
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