We are at an exciting time in the development of therapies for DMD with clinical trials in progress, or planned using several different approaches. The future has never been brighter for DMD boys.
Professor Dame Kay Davies, Oxford University
Back in 2013 we published the first Duchenne specific research strategy. The progress in Duchenne research since then, led by organisations like Action Duchenne, has meant that we have had to keep adapting our strategy to take advantage of the opportunities that have arisen, as well as overcome certain hurdles.
Our new Research Strategy published in late 2017 set out our research funding priorities over the next 3 years, with a focus on:
- DMD Registry – Strategically investing in both the capacity that underpins the UK DMD Registry, but also in the database itself. With the aim of being at the forefront of facilitating clinical trials and other translational research
- Innovation – We will be co-funding novel ideas by Duchenne healthcare professionals who apply, and will publish their successful results
- Outcome measures and natural history data
- Investing in tissue collection and biobanking – Establish a viable platform and shared bank of biological specimens for the use of the community
- Standards of Care – Continuing our long-standing involvement in supporting the best possible multidisciplinary standards of care, across the age spectrum
The community has begun to realise that individuals with Duchenne are living longer, with the need to tailor treatment for both ambulant and non-ambulant individuals being more important than ever. The new Action Duchenne strategy reflects this change supporting innovative research and current clinical trials, with a real focus on improving the quality of life of all those affected.
Benjamin James, Member of Action Duchenne Research Committee and living with Duchenne.
Further information
For more information about our research strategy, call us now on 07535498506 or email info@actionduchenne.org
