Posted by Michael Wonder on 30 Sep 2022
NICE review does not recommend routine funding for Duchenne muscular dystrophy gene therapy ataluren in draft guidance
30 September 2022 - NICE has today published draft guidance for public consultation which does not recommend ataluren (Translarna, PTC Therapeutics) for treating Duchenne muscular dystrophy caused by a nonsense mutation.
Today’s decision follows NICE’s commitment to re-evalutate ataluren and decide whether it should be available for routine funding on the NHS following a 6 year period during which it was available under a managed access agreement.
