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active, not recruiting

Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping

key information

study id #: NCT03218995

condition: Duchenne Muscular Dystrophy

status: active, not recruiting


This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.

intervention: Eteplirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03218995

last updated: April 08, 2020