The Wilson family sat in their living room playing with their son Aadi who has SMA Type 2
 Professor Dame Kay Davies in her laboratory at the University of Oxford working with two researchers
Two delegates share laughing together in the coffee break at the Muscular Dystrophy UK 2019 conference
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NICE publishes draft guidance on access to Duchenne muscular dystrophy treatment Translarna

NICE has published draft guidance stating that although Translarna (also known as ataluren) is clinically effective and an innovative treatment, there is doubt as to its cost effectiveness. NICE’s draft recommendation is that people currently receiving Translarna should continue to have access to it until they and their NHS clinician consider it appropriate to stop, but that it shouldn’t be provided for new patients after January 2023. This continuing access for existing patients is welcome, but we are very disappointed by this draft decision regarding new patients and are urging NICE and PTC Therapeutics to work together to address the concerns raised about cost effectiveness.
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