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Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

key information

study id #: NCT02286947

condition: Duchenne Muscular Dystrophy

status: completed


The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced 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/NCT02286947

last updated: April 08, 2020