Eteplirsen Proven Effective for Duchenne Muscular Dystrophy
The study assessed eteplirsen's safety, tolerability, pharmacokinetic profile and ability to restore dystrophin expression.
AVI BioPharma, a developer of RNA-based therapeutics, has released positive Phase Ib/II study results of eteplirsen, an exon-skipping therapy for the treatment of Duchenne muscular dystrophy.
The genetic muscle-wasting disease is caused by the absence of functional dystrophin, an essential muscle protein.
The 19-patient, 12-week, six-dose cohort study assessed eteplirsen's safety, tolerability, pharmacokinetic profile and ability to restore dystrophin expression. The study also showed that eteplirsen induced exon 51 skipping in all cohorts, and dystrophin protein expression was observed in a dose-dependent manner.
Francesco Muntoni, head of the Dubow
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