1. Prosensa Achieves Orphan Drug Status on its Entire

    Posted on January 29, 20132:39 pm

      Leiden, The Netherlands – 29 January 2013 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, announced it has achieved orphan drug designation in the EU and the US for all of its compounds for the treatment of Duchenne muscular dystrophy (DMD). The European Medicines Agency Read more »

  2. GSK Exon 51 Trial Update

    Posted on January 10, 20132:25 pm

    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 Read more »