GSK Exon 51 Trial Update
GSK has sent out this update on their Duchenne trial. The last patient has been recruited within the drisapersen US Phase II clinical study [www.clinicaltrials.gov NCT01462292]. This exploratory study aims to assess the safety, efficacy and pharmacokinetics of two doses of drisapersen in the treatment of ambulant boys with DMD who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of all boys with DMD). The Primary Outcome Measure is mean change from baseline of the distance that the patient can walk in 6 minutes (measurement performed through the standardised 6 Minutes Walk Distance Test- 6MWDT) following the 24 week treatment period, and the study also has a 24 week post-treatment phase for continued monitoring. The study is expected to complete in November 2013, with study results available in early 2014. The results will be presented at the first appropriate medical conference.