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Evidence-based recommendations on ataluren (Translarna) for Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people 2 years and over who can walk.
Routine funding recommended for Duchenne muscular dystrophy gene therapy ataluren
NICE has today published final guidance which recommends ataluren (also called Translarna and made by PTC Therapeutics) for treating Duchenne muscular dystrophy (DMD) caused by a nonsense mutation.