Aartsma-Rus A, Krieg AM. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga. Nucleic acid therapeutics 2017;27:1-3
On 19 September 2016 the first AON for DMD was approved by the FDA (Eteplirsen; PMO-backbone; against exon 51). In this commentary the clinical data of Eteplirsen and the implications of this approval are discussed.