Translarna Now Available for Duchenne in England

Post Date: 
11 July 2016

The National Health Service (NHS) England and PCT Therapeutics have negotiated a managed access agreement (MAA) for Ataluren (Translarna) for ambulatory patients of the age of five years and older with nonsense mutation Duchenne muscular dystrophy (nmDMD).

The agreement between the two parties provides patient access to Translarna which previously received a positive recommendation from the National Institute for Health and Care Excellence (NICE) who will help provide financial guidance for the MAA. The commercial agreement consists of five years and a managed access scheme for patients, which includes an outcomes-based incentive for the manufacturer along with a mechanism to monitor how well the Translarna has worked in practice. NICE guidance and plans are being put in place to allow treatment to commence in designated specialist centers soon afterwards.

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