Access to treatments

Duchenne UK expands the DMD Hub to give DMD patients in London and the South-East more opportunities to access research

Duchenne UK is delighted to announce a significant expansion of its ground-breaking DMD Hub project -- bringing on board Evelina Children’s Hospital to the DMD Hub and opening up the potential for more people with Duchenne Muscular Dystrophy (DMD) in London and the South East of England to have access to the most exciting areas of research into the condition.

Great Ormond Street Hospital conducts many clinical trials for DMD for the London area, but has limited capacity. By investing in Evelina, Duchenne UK aims to expand trial opportunities for other patients living in London and the South-East.

Vasantha Gowda (pictured with Duchenne UK co-founders, Emily Crossley and Alex Johnson), Consultant Paediatric Neurologist at the Evelina London Children’s Hospital said:

We are very grateful to Duchenne UK for funding these two posts, and are excited to be joining the DMD Hub. We have an excellent team at Evelina and this funding will help us to expand the number of clinical trials we are already running as well as future-proof our clinical trial capacity.

The first year’s funding is being provided by our partner charity, Chasing Connor’s Cure. When founder Matt Crawford’s son Connor (pictured below) was diagnosed in 2016 he was keen for him to be treated at a hospital with research facilities. However, the only hospital near him, Great Ormond Street Hospital (GOSH), was already at capacity.

Matt Crawford said: 

“Research trials provide hope to the DMD community; hope is what every family craves.  Unfortunately, when there was no space for Connor at GOSH, one of only two research facilities at the time, we were devastated and perplexed.  How can there be so many trials, yet no resources to support them?”

Our co-founders Alex Johnson and Emily Crossley were already aware of the capacity problems at the only two hospitals in the UK able to run DMD clinical trials. So, in 2015 the DMD Hub was created and since then Duchenne UK has worked determinedly to expand capacity at sites all over the UK.

Duchenne UK has invested £1.79 million into the DMD Hub. 194 boys have been recruited to take part in clinical trials since the DMD Hub started.

Matt Crawford told us, 

Connor was instead put under the care of Evelina London Children's Hospital and we soon appreciated the good work Evelina put in and have seen the benefits through Connor’s care.  When Duchenne UK launched the DMD Hub project, we immediately put Evelina forward as a centre that would benefit from investment into delivering research trials, benefitting Connor and other boys that visit the hospital.

We are immensely grateful to Duchenne UK and our fundraisers to be in a position to provide Evelina with the resources to establish themselves as a research centre.  This represents a lot of hard work from all involved, that provides a tangible result for all the support we have received since launching Chasing Connor’s Cure in 2016.

The £193,487 grant to Evelina London Children's Hospital will fund a clinical research fellow and a physiotherapist to support the team’s work on clinical trials.

Through the DMD Hub, we are funding 22 posts, including doctors, nurses, physiotherapists and support staff, at DMD Hub hospital sites all over the UK to help the sites run more clinical trials to give all patients with DMD in the UK access to potential new treatments. We also provide sites with mentoring and training and help connect them with pharmaceutical companies looking for sites to run their trials.

To find out more about the DMD Hub and to search for clinical trials visit DMDHUB.ORG

To receive clinical trial updates directly into your inbox, Join the Hub. 

We need your help to keep funding the DMD Hub so we can bring promising research straight to patients DONATE NOW


What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy 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. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

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:

Visit www.duchenneuk.org

Molly Hunt – Head of Communications, Duchenne UK E: [email protected]

Published on 6 May 2019

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