16 October 2015 - In draft guidance produced by its Highly Specialised Technologies programme, NICE has asked for further clarification from PTC Therapeutics on the size of the benefit its drug ataluren (Translarna) provides in the treatment of a type of Duchenne muscular dystrophy (DMD). NICE has asked the company to provide the results of a confirmatory study of ataluren as soon as possible. The draft guidance also asks the company to provide further justification for the cost of the drug.

For more details, go to: http://www.nice.org.uk/news/press-and-media/nice-asks-for-further-evidence-for-the-benefits-of-a-new-treatment-for-duchenne-muscular-dystrophy-to-justify-its-very-high-cost