FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

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

Aartsma-Rus_2017_Nucleic Acid Ther. Feb;27(1);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.

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