Access to treatments

Duchenne UK awards £41,151 through the DMD Hub to Bristol Royal Hospital for Children, to help expand clinical trial capacity

As part of our commitment to expanding clinical trial capacity throughout the UK, we are delighted to announce that Bristol Royal Hospital for Children is joining the DMD Hub as a Hub site.

Duchenne UK has awarded £41,151 to the trust to recruit a Clinical Trial Coordinator. This funding is part of Duchenne UK and the DMD Hub’s mission to increase clinical trial capacity in the UK.

The grant will support a 3-year Clinical Trial Coordinator post at Bristol Royal Hospital for Children, using the DMD Hub’s innovative tapered funding model. This staged funding model ensures posts introduced to the NHS Trusts by the DMD Hub are sustainable once the grant has ended.

In addition to funding, the DMD Hub will also offer Bristol Royal Hospital for Children support and expertise to help work with industry to run more DMD trials.

Bristol are already confirmed as a site for the Wave Phase 1 study and the Catabasis POLARIS study. Visit the DMD Hub website for more information about ongoing trials at Bristol. 

Dr Anirban Majumdar, Consultant Paediatric Neurologist at Bristol Royal Hospital for Children, said: 

 “We here are delighted to have received this funding from the DMD Hub for the post of Neuromuscular DMD research coordinator. This post ensures that all the boys and young men with DMD, under the care of the children’s hospital, who are able to participate in research trials will now have a dedicated person who would be able to coordinate all the research related activities. This will go a long way to help increase the clinical trial capacity in Bristol and the South West ensuring that all with DMD have access to research nearest to where they live.”

Alex Johnson, co-founder of Duchenne UK said:

"We are very pleased to be supporting Dr Majumdar and his team to help them deliver more clinical trials. Duchenne UK has now committed £2million to the DMD  Hub and are pleased to see the number of trials increasing in the UK.”

The DMD Hub is a network of trial sites with trained staff that are funded to carry out clinical trials for DMD. Our ambition is that everyone in the UK diagnosed with DMD is given the opportunity to take part in research.

Make sure you have JOINED THE HUB to stay up-to-date with clinical trials in the UK.

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 DMD 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. 

For more information: visit www.duchenneuk.org or email Molly Hunt, Communications Manager: [email protected]

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 DUCH10 £10 to 70070  

Published on 20 December 2018

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