Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol

Summary

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.

Timeline

Start

2014-01

Primary completion

2014-01

Completion

2015-07

Outcome

Outcome not reported

Stopped (Enrollment): “Rights for the further development of drisapersen have been transferred to Prosensa. Therefore this study has been cancelled before enrollment.”

Drugs

Indications