Duchenne UK Impact Report 2023
A landmark year of achievement and impact
A landmark year of achievement and impact
Duchenne UK has one clear aim – to end Duchenne muscular dystrophy (DMD); a severe muscle-wasting disease, diagnosed in childhood.
As the leading DMD charity in the UK, we want to bring an end to Duchenne’s devastating impact. We’re going further to find effective treatments by funding ground-breaking medical research, and we’re doing it faster, by overcoming the barriers in the way of getting treatments to this generation of patients.
We are also here to support every family and to make sure they receive the best care.
We work with scientists, the pharmaceutical industry, the NHS and families to make real change happen.
Together, we will end Duchenne.
Our impact in treating DMD could not have been achieved without our wonderful community of funders.
This year has seen a breakthrough – the first ever drug approved for use on children with DMD, Vamorolone, designed specifically for their condition. It’s reaching patients because Duchenne UK and our partner charities funded the early-stage clinical research to test this drug in patients, when no-one else would.
When we first met in 2012, we were broken by a diagnosis that had robbed our sons of their future. But through that pain, we never lost sight of our goal; to claw some of that future back for our sons and those in families like ours.
We were told the standard medication for children with DMD, steroids, would keep them independently mobile for longer, but with harmful side effects. We didn’t think this was good enough, and invested in finding better treatments.
Now Vamorolone has been approved for use in people with DMD in the US and Europe, and we hope the UK will follow soon.
The way Duchenne UK has helped achieve this significant milestone towards an improvement in how DMD is treated is a perfect demonstration of what Duchenne UK is about. A new, better drug, successfully coming to market for children with DMD, is proof that our charity has impact, and that we get results for our community of families and the people who so generously support our cause through fundraising.
And that’s not all.
When our sons were diagnosed, we were told gene therapy would not be ready in their lifetime. We disagreed. We were seed funders of Solid Biosciences, a start-up established to pursue this new technology, and boys are being dosed with gene therapy in clinical trials taking place right now in the UK.
We learnt that patients with DMD were dying too young and their quality of life negatively impacted because they were not getting the right care. DMD Care UK is driving best practice across all medical disciplines so that everyone living with DMD in the UK has access to the best care. Following the publication of our cardiac guidelines in 2022, this year we launched new guidance on how DMD affects bones, helping parents know exactly what children and adults with DMD need.
We’re driving technological advances, for the benefit of DMD children and others losing their mobility. We’re leading a collaborative R&D project to develop the first ever arm assist device to support arm function. Its third version is undergoing testing now. It’s a project that puts the needs and aspirations of DMD children at its heart, with the potential to bring benefit to other people with arm mobility issues.
All of this couldn’t happen without the incredible, selfless support of our funding partners, our Family & Friends Funds and our individual fundraisers. We offer our sincerest thanks to every one of them, whose support means we can continue on our mission to find treatments for DMD.
With love and thanks,
Emily Reuben OBE and Alex Johnson OBE
Duchenne UK founders
Alex and Emily each received an OBE in the King’s Birthday Honours list, for their work to find treatments and improve care to transform the lives of everyone affected by DMD.
Krishnan Guru-Murthy, Chair of Duchenne UK, said, “Alex and Emily have dedicated their lives to transforming the scientific landscape and bringing hope to families with Duchenne worldwide, while living with the heart-breaking reality of having boys with DMD. They are truly deserving of their honours.”
Krishnan Guru-Murthy (Chair)
Caroline Ayling
Cecilia Crossley
Nick Crossley
Chris Harris
Harriet Moynihan
Andrew Nebel MBE
Peter Williams
Alex Bilmes
Andy Farrell OBE
Ben Levine
Clare Runacres
Jonathan Whitworth
Kris Radlinski MBE
Mary Nightingale
Owen Farrell
Peter Serafinowicz
Peter and Laura Williams
Sara Parker Bowles
Sarah Alexander
Find out more about our research and access to new treatments impact in 2023