Sarepta Therapeutics announced that the Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

RELATED: Eteplirsen NDA Submission Completed for Duchenne Muscular Dystrophy

Eteplirsen is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. It uses Sarepta’s novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13% of the total DMD population.

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