The impact of our research
Since our founding, Duchenne UK has invested millions into research and infrastructure. Here’s what that means for families living with Duchenne muscular dystrophy.
Measuring success
We measure success by impact—not just publications. Every project we fund is chosen for its potential to deliver real benefits to patients.
Our investment
in research
34
Posts in the Duchenne
Hub UK Clinical Trial Network
106
Projects funded
£15M
£15M of which £3.3M towards research infrastructure and the rest largely towards therapeutic development
The impact of our funding
How we helped Vamorolone become a reality for the Duchenne community
Phase 1 - Funding
£750,000 of funding by Duchenne UK, Joining Jack and Duchenne Research Fund bridged the valley of death in clinical research by funding Phase 1 of research into vamorolone
Phase 2 - Clinical Development
We worked with the company to select the outcome measures for the Phase 2 and Phase 3 trials, which gave clinical meaningfulness to the data
Phase 3 - Clinical Development
We supported Professor Michela Guglieri, from the John Walton Muscular Dystrophy Research Centre at Newcastle University, who was the study chair of Phase 2b, and the UK hospital sites which delivered it
Phase 4 - Collaboration
We helped Newcastle University and ReveraGen, the company developing vamorolone, win a 6 million euro investment from the European Union to undertake Phase 2 research into it by supporting their application
Phase 5 - Value Assessment and Access
Our Project HERCULES provided crucial evidence about the impact of Duchenne to build the case to National Institute for Health and Care Excellence (NICE) for patient access to it
Phase 6 - NHS Adoption
Through Duchenne Care UK, we are supporting vamorolone’s implementation in the NHS, to ensure that every patient who wants it can access it in a timely and safe way. We published a new Duchenne Care UK family guide on steroids in 2025
