Duchenne UK invests £1.25 million into new research of treatments for DMD
- Four projects will receive funding from our DMD INSPIRE Major Grant Call
- These projects explore ways to build new muscle, tackle fibrosis and bring treatments to patients as quickly as possible
- Duchenne UK has now committed £11.5 million to medical research to advance treatments for DMD
Duchenne UK is pleased to announce the four recipients of our DMD INSPIRE Major Grant Call. £1.25 million will be invested in developing treatments for Duchenne Muscular Dystrophy (DMD).
We launched our grant call to find answers to the big problems we need to tackle to END DUCHENNE:
- How can we treat the root cause of the disease?
- How can we remove fibrosis?
- How do we help muscles regenerate and so halt progression of DMD, or reverse it?
The four projects look at different ways to build new muscle, tackle fibrosis and regenerate muscle. All are focussed on bringing new treatments to patients in as short a time frame as possible.
They include supporting pre-clinical work for a clinical trial to test stems cells, and testing existing medicines in combination. From a humble vitamin to the advanced pluripotent stem cells, we are exploring all aspects of DMD, building up our understanding of the disease and how best to combat it.
Duchenne UK is now funding more than 60 projects worldwide. These range from seed funding for gene therapy, to funding clinical trials, to expanding clinical trial capacity so all patients can access research. We are committed to accelerating the search for treatments for this generation of patients living with DMD.
Full details of the projects can be found below
- Creating new muscle by using stem cells
- Tackling fibrosis with repurposed medicines
- Investigating the benefits of Vitamin B3 (Nicotinamide Riboside)
- Helping muscle regeneration with repurposed medicines
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 is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.
We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.
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 and interview requests:
Molly Hunt – Head of Communications, Duchenne UK E: email@example.com
Published on 8 February 2019Share this article Categories DMD research