Trial ID or NCT#



not recruiting iconNOT RECRUITING


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.

Official Title

A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study

Eligibility Criteria

Ages Eligible for Study: Older than 5 Years
Sexes Eligible for Study: Male
Accepts Healthy Volunteers: No


Yuen So, MD, PhD
Yuen So, MD, PhD
Neuromuscular neurologist, Neurophysiologist
Professor of Neurology

Contact us to find out if this trial is right for you.


Shirley Paulose
(650) 724-3792