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Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
Personal take on this article: This study evaluates the safety, tolerability, and pharmacokinetics of eteplirsen in boys aged 6 to 48 months with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO), is designed to restore dystrophin production. The open-label, dose-escalation trial involved 15 boys divided into two cohorts: one group aged 24 to 48 months and another aged 6 to …
