Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
To continue evaluation of the long term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD) Three year progression of eteplirsen treated patients was compared to matched historical controls (HC)
Annals of neurology