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completed

Study of Eteplirsen in DMD Patients

key information

study id #: NCT02255552

condition: Duchenne Muscular Dystrophy (DMD)

status: completed

purpose:

The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).

intervention: eteplirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT02255552

last updated: July 06, 2020