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Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
This is a single arm, open-label continued access protocol of drisapersen for the treatment
of male subjects with Duchenne muscular dystrophy (DMD) having dystrophin mutations
correctable by drisapersen-induced DMD Exon 51 skipping. The purpose of this continued
access protocol is to offer pre-approval access to drisapersen for the treatment of subjects
with DMD who previously participated in eligible drisapersen studies. The protocol will
collect safety data required to assure subject safety and periodic efficacy data on muscle
function.