Translarna (also known as ataluren) has been developed to treat children whose Duchenne muscular dystrophy is caused by a ‘nonsense’ mutation. Translarna is the first drug to address an underlying genetic cause of muscular dystrophy to be recommended for use on the NHS in England. It is available for children who are:  

  • aged five years and over
  • are still able to walk – 10 steps unaided

Currently, Translarna is not licensed for younger children or children who have lost the ability to walk. More clinical trials will be needed to assess the efficacy and safety of Translarna in these groups of individuals.