NICE has recommended funding on the NHS in England for Translarna.

NICE has recommended funding on the NHS in England for Translarna.

This historic decision would make available on the NHS the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy.

Translarna would treat boys whose Duchenne is caused by a nonsense mutation, who are aged five years and over and who are still able to walk.

In order to give time for NHS England and the drug company, PTC Therapeutics, to agree an final acceptable price, NICE has set July as the date where its recommendation will become final. We’ll then be pushing NHS England to implement this decision in order to get the drug to boys in clinic as quickly as possible. Read More