Access to treatments

Duchenne UK funds DMD Hub Manager post for a further 4 years

Duchenne UK is committed to the ongoing success of the DMD Hub and today announced a further four years of funding for the DMD Hub Manager role, bringing total investment in the DMD Hub to £4.3million.  

The DMD Hub is one of Duchenne UK’s flagship projects and has successfully expanded capacity for clinical trials for Duchenne muscular dystrophy (DMD) treatments in the UK. In the first four years of the project, Duchenne UK and its partner charities invested £2.7m in the DMD Hub, with an additional £1.6m committed over the next 4 years.  

The DMD Hub is a collaboration between Duchenne UK, the John Walton Muscular Dystrophy Research Centre (JWMDRC) in Newcastle, and Great Ormond Street Hospital London. It was set up to expand clinical trial capacity and expertise and stop clinical trials being turned away from the UK. The DMD Hub has funded 30 posts, which ranges from research nurses to physiotherapists, from clinical research fellows to trial coordinators.  

The project team also developed the Clinical Trial Finder to help patients access information on DMD clinical trials in the UK. The tool includes information on each trial taking place, including selection criteria, age range, a lay summary and details of the sites that are recruiting. It was cited as an excellent example of digital infrastructure in a recent report by the Association of Medical Research Charities.  

Thanks to the DMD Hub, to date, more than 300 boys have been recruited to clinical trials who otherwise may not have been. Additionally, 11 UK hospitals are conducting 10 trials into potential treatments for DMD.  

You can read more about the achievement of the DMD Hub has had in our DMD Hub Impact Report 2020.  

Emma Heslop is the DMD Hub Manager and is key to the success of this project. Working closely with the Duchenne UK team, she ensures patients are appropriately represented as key stakeholders in all aspects of the DMD Hub. As part of the team at the JWMDRC in Newcastle, she has direct access to clinicians and industry partners through the relationships she has cultivated over the years.  

As the main point of contact for all companies with trials coming to the UK, she also promotes the UK as an attractive place to run DMD trials and works with them to support site selection, set up and patient recruitment. 

Her role also involves coordinating the staff networks for clinical trial coordinators, nurses and lead clinicians, which include staff from across all eleven DMD Hub sites. These networks promote communication between the different sites so that they can share best  practice and expertise. They also highlight any training needs, which the DMD Hub can then help to deliver.  

Her workplan for the next 4 years includes several exciting projects, which will have a significant impact for the DMD community:  

  1. Developing an operating procedure, adopted by all UK DMD Hub sites, to centrally coordinate patient recruitment to DMD trials 
  2. Conducting a Patient Preference survey, in collaboration with PPMD, to better understand patients and caregivers perceptions of the benefits and risks of taking part in gene therapy trials 
  3. Conducting an Institutional Readiness survey to assess which hospitals in the UK are able to run gene therapy trials, and then to deliver a gene therapy licenced product. The survey will also identify barriers to hospitals hosting trials, which the DMD Hub will work to address.  
  4. Facilitating the implementation of the DMD Care UK Project across all DMD Hub sites 
  5. Engaging with key initiatives in the UK facilitating gene therapy such as the Cell and Gene Therapy CATAPULT and the NA-ATTC (Northern Alliance for Advanced Therapies Treatment Centre) 
  6. Working with the MHRA (Medicines and Healthcare products Regulatory Agency) to ensure that post-Brexit, the UK remains an attractive site for trials

The cost of funding the DMD Hub manager post for another 4 years is £318,163 and is crucial for the continuing success of the DMD Hub.   

We would like to thank our charity partner Alex’s Wish, and the following Family Funds for contributing to this project: Jack’s Mission, Help Harry, Jacobi’s Wish, Jack’s Aim, Action for Arvin, Ralphy’s Fund, Henry’s Hurdles, Ben vs Duchenne, Moving Muscles for Marcus. 

Published on 19 May 2021

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