If you or your child has experience of taking Raxone / idebenone, please help us by filling in our survey: https://www.surveymonkey.co.uk/r/raxone

30th July 2020

The National Institute for Health and Care Excellence (NICE) is consulting on a new medicine called idebenone which could slow down the decline in respiratory function for patients with DMD. 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.

Idebenone, made by Santhera Pharmaceuticals, is currently available to a small group of patients in the UK through the Early Access to Medicines Scheme (EAMS), and is called Raxone. We really want to hear from these patients!

Duchenne UK is working with DMD Pathfinders, Joining Jack, Muscular Dystrophy UK, Action Duchenne, Alex's Wish and Duchenne Family Support Group. We are asking the DMD community to help us reach as many people as possible with experience of taking Raxone to fill in this survey.

If you or your child takes Raxone for DMD, please complete this survey by 19th August. If you know someone who may be taking Raxone, please pass this survey on to them: https://www.surveymonkey.co.uk/r/raxone

Click here to find out more about Raxone and EAMS.


NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK has one clear aim – to end Duchenne, a devastating muscle-wasting disease. As the leading Duchenne charity in the UK, we connect the best researchers with industry, the NHS and families to challenge every stage of drug development to make the incurable, curable. Together, we will find treatments and cures for this generation of patients with Duchenne.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org