Thursday 17 February 2011

GSK and Prosensa announce start of Phase III study of investigational Duchenne Muscular Dystrophy medication

GSK and Prosensa announce start of Phase III study of investigational Duchenne Muscular Dystrophy medication: "GlaxoSmithKline (GSK) and Prosensa today announced that the first patient has commenced treatment in the Phase III clinical study investigating GSK2402968 (`968), in ambulant boys with Duchenne Muscular Dystrophy (DMD), who have a dystrophin gene mutation amenable to an exon 51 skip (up to 13% of boys with DMD). Commencement of this study confirms previously announced plans to progress this asset into Phase III.
This randomised, placebo controlled study will enrol 180 patients, from up to 18 countries, and is currently the most advanced ongoing study for this rare, severely debilitating, neuromuscular disease.
The study is designed to assess the efficacy and safety of GSK968 6mg/kg, once weekly, compared to placebo, for 48 weeks in ambulant boys over 5 years of age with DMD The primary efficacy endpoint is a measure of muscle function using the six minute walking distance test."

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