In draft guidance published on Friday, it emerged NICE does not recommend PTC Therapeutics’ Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense
A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics' Translarna for the first time.