The National Institute for Health and Care Excellence (NICE) is consulting on a potential new drug called Idebenone (or Raxone) which may slow down the decline in respiratory function for patients with Duchenne.
Your response will help inform us about patients’ experiences of taking Raxone, which will help NICE make a decision about whether the medicine should be funded and made available on the NHS in England if it is approved for use in Duchenne.
Please share your experiences
We are asking the DMD community to help us respond to the consultation: if you or your child has experience of taking Raxone please fill in our online survey by 19th August. If you know someone who may be taking Raxone, please pass this survey on to them.
Action Duchenne is working with Muscular Dystrophy UK, Duchenne UK, DMD Pathfinders, Joining Jack, Alex’s Wish and Duchenne Family Support Group to respond to the consultation.
Why take the survey?
The consultation process is open to patient organisations who are asked to collect evidence from patients about understanding what it’s like to live with DMD and to help NICE understand what impact this medicine may have. At the end of the process NICE will make a decision about whether the medicine should be funded and made available on the NHS in England.
Raxone, made by Santhera Pharmaceuticals, is currently available to a small group of people in the UK through the Early Access to Medicines Scheme (EAMS). If you are part of this small group, your opinions are vital and we really want to hear from you!
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