Fig. 2

Exondys 51™ excludes dystrophin exon 51 and corrects the DMD reading frame, therefore reducing disease severity in the most common type of DMD mutation. a Normal splicing of DMD exons 49–52. b Exon 51 is deleted from the DMD transcript as the result of a mutation at affect about 13% of DMD patients. Exondys 51 alters splicing of exon 51, removing it from the DMD transcript, restoring the reading frame and reducing disease severity, reflecting a Becker MD phenotype