NICE recommends ataluren for Duchenne muscular dystrophy
The UK National institute for Health and Care Excellence (NICE) has recommended PTC Therapeutics’ Translarna (ataluren) for treating children aged 5 and over with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation. Commenting on the recommendation, Sir Andrew Dillon, chief executive of NICE, said: “Duchenne muscular dystrophy caused by a nonsense mutation is a cruel disease that currently has few treatment options. Ataluren is a medicine that for the first time is aimed at the root cause of the disease and has the potential to offer benefits to people with the condition and their families.”
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