Our report – Transforming our rare reality
Our new report, Transforming our rare reality, will give you a complete picture of what it is like to live with DMD in the United Kingdom in 2024.
It reports on the gaps in treatment, care and support, and sets out our vision and our roadmap for change. In it, we take stock of the progress that has been made in the 13 years since we set up Duchenne UK.
When our sons, Eli and Jack, were first diagnosed, we were bereft. Instead of being scooped up by a protective ring of health and social care, we realised, from the beginning, that we were going to have to fight for everything. A fight to access social care support, a fight to get access to the best standards of care, a fight to get the right equipment.
We were facing unimaginable odds and battling a relentless and horrible disease. But there was almost nothing to help us. Very limited treatments, little support and very little care – and no hope.
That was 13 years ago, and a lot has changed since then for people living with DMD in the UK.
A child diagnosed with DMD has a far better prognosis because of improved treatments, better care, and more support.
Healthcare professionals covering many disciplines have played a key role in this progress.
Our amazing community
So too has an incredible community of people personally affected by DMD. There are the amazing DMD Family and Friends Funds that raise hundreds of thousands of pounds to support our work.
There are the brave families and sons who sign up for research – who fill in questionnaires and who take part in clinical trials – many of which do not reach their primary endpoints of success.
Our partner charities: Joining Jack, which Alex and Andy Johnson set up after their son Jack’s diagnosis with DMD. Many other brave families have done the same and have now joined up with us – because together we are stronger. Alex’s Wish, which was set up by Emma Hallam, whose son Alex has DMD. The Duchenne Research Fund that was set up by Kerry and Doron Rosenfeld after their son Gavriel was diagnosed with DMD; Chasing Connor’s Cure that Matthew and Emma Crawford set up following their son Connor being diagnosed with DMD. Duchenne Now, which Tony Levene set up when his son Joey was diagnosed with DMD.
And it’s a community of driven parents fighting to change the world. And we are lucky to partner with those people.
Dr Janet Hoskin, whose son Saul has DMD, has written a book to guide people on DMD, and runs DMD education support programme Decipha with her husband Nick Catlin.
Fleur Chandler, whose son Dominic has DMD, set up Project HERCULES with us to build the case for approval of new DMD treatments.
Annie and Ilan Ganot, whose son Eytani has DMD, set up Solid Biosciences, a life science company focused on advancing genetic medicines for neuromuscular and cardiac diseases, and who helped kickstart the development of Elevex, which is our new technology to assist arm movements.
The wonderful Manoj and Sejal Thakrar, whose son Shiv had DMD. They set up the Family and Friends Fund ‘Smile with Shiv’, and Sejal, whose background is data analysis, undertook some of the research that went into this report.
You can download the report here. Thank you for reading it, and we hope to work with you on our framework for change.
Emily Reuben OBE, Chief Executive, Duchenne UK, and founder of Duchenne UK
Alex Johnson OBE, Chief Executive, Joining Jack and founder of Duchenne UK
