Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy


OBJECTIVE 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). METHODS Ambulatory DMD patients who were ≥7… (More)
DOI: 10.1002/ana.24555


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