26th Feb 2018

Raxone (also known as Idebenone) is a drug produced by Santhera Pharmaceuticals. The active substance in Raxone is called idebenone, which is believed to improve energy production in muscle cells. Raxone is believed to help slow down the decline in respiratory function for DMD Patients by slowing the rate at which the respiratory muscles weaken. 

Under the Early Access to Medicines Scheme, Santhera are providing the drug to the NHS free of charge for patients who qualify for it. The company will continue to collect data on the effectiveness and safety of Raxone to support their application to the EMA.

In partnership with Action Duchenne, Muscular Dystrophy UK, the Duchenne Family Support Group, and DMD Pathfinders, we have produced two guides to educate and answer questions on Raxone and the Early Access to Medicines Scheme (EAMS) process.
 
The first guide is for families: Raxone- a guide for the Duchenne community
 
The second for healthcare professionals: Raxone – a guide for clinicians
 
If you have any questions, please get in touch with us [email protected]

NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy? 
Duchenne Muscular Dystrophy 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 is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

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:

For more information and interview requests:
Visit www.duchenneuk.org
Molly Hunt – Communications Manager, Duchenne UK 
E: [email protected]