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FDA 'breakthrough' for GSK/Prosensa DMD drug drisapersen rattles Sarepta

This article was originally published in Scrip

Executive Summary

GlaxoSmithKline could not wait to tell investors and patients on 27 June the London drug maker had been informed by the US FDA – at least verbally – the agency had granted breakthrough therapy designation to drisapersen (GSK2402968/PRO051) as a treatment for Duchenne muscular dystrophy (DMD), a rare severely debilitating neuromuscular disease caused by mutations in the dystrophin gene, which is essential for muscle function.

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