This year the National Institute of Health and Care Excellence (NICE) is conducting its final appraisal of Translarna (also called ataluren).
This process will decide whether the treatment is made available on the NHS once the current Managed Access Agreement (MAA) comes to an end in January 2023.
Action Duchenne worked in partnership with Muscular Dystrophy UK and with families affected by Duchenne muscular dystrophy to establish access through the MAA six years ago and we are committed to doing so again as we seek permanent approval.
NICE will not make a final decision until later this year; but we need your support now to ensure that they hear the views and understand the experience of the Duchenne community.
Take our survey
Action Duchenne and Muscular Dystrophy UK are working together to gather the views of the Duchenne community to ensure that they are represented in the appraisal process.
Tell us about your experience of Translarna by completing this survey by 25th February.
The information you provide will feed directly into the joint submission being made by Muscular Dystrophy UK and Action Duchenne to NICE.
Join us on Zoom at 12-1.30pm on 22 February
We are delighted to announce that Brad Groves, Associate Director of Managed Access at NICE, has agreed to join us to explain the appraisal process; what this means for the current MAA and to answer questions from the Duchenne community.
The session will also be a chance to learn more about how you can get involved; hear from people involved in the first campaign for access and to discuss your experience of Translarna, which will help Action Duchenne and Muscular Dystrophy UK finalise our joint response to the appraisal consultation.
Becoming a foster carer
