UK – NICE recommends PTC Therapeutics’ Translarna reimbursement

The NICE recommendation followed data from clinical trials and real-world evidence

PTC Therapeutics has announced that the National Institute for Health and Care Excellence (NICE) has issued a final evaluation document recommending Translarna.

It involves reimbursement and wider use across the NHS in England and Wales. Translarna – also known as ataluren – is the only approved treatment for patients with nonsense mutation Duchenne muscular dystrophy, aged two years and older, who have the ability to walk…