We know that funding research alone is not enough to ensure that DMD patients can access the treatments they need. That's why we're going further, and breaking down barriers at every stage of drug development.
DMD is a progressive disease, and we know that this generation of boys can't wait. That's why we've launched innovative, collaborative projects to increase the chances of treatments being tested in clinical trials, approved and made available to those that could benefit from them as soon as possible.
We launched Project HERCULES in (HEalth Research Collaboration United in Leading Evidence Synthesis) in 2017: a groundbreaking, global project which brings pharmaceutical companies together to increase the chances of new DMD treatments being approved and made available to patients.
Pharmaceutical companies wanting to introduce new treatments for patients are asked to submit evidence of clinical and cost effectiveness to Health Technology Assessments (HTA) agencies and payers. We set up Project HERCULES in response to the challenges and delays in the HTA of the first licensed treatment for DMD.
The projects aims to improve the evidence base and tools available to pharmaceutical companies, so that their treatments can be better assessed.
Project HERCULES has already delivered:
The DMD Hub is a network of hospital sites with trained staff which are funded to carry out clinical trials for DMD.
We set up the DMD Hub to increase the availability of clinical trials in the UK and ensure more DMD patients could take part in research into the most promising treatments. Before the DMD Hub was launched, clinical trials for DMD were only conducted at Great Ormond Street Hospital (GOSH) in London and the Newcastle-upon-Tyne Hospital. These sites were at full capacity and turning away trials from drug companies.
Thanks to the Hub, there are now 11 hospital sites in the UK running DMD trials. We fund the staff needed to run trials effectively, support sites to increase their capacity, and work with pharmaceutical companies to help them set up trials in the UK.
Patient organisations play a pivotal role in helping regulators and policy makers understand the realities of living with DMD. We need to engage with them early in the drug development process to ensure that when drugs do become approved for use, the NHS can afford to pay for them, and understands the true value of these potential treatments for patients and families living with DMD.
Duchenne UK works with regulators on a number of issues, from how to speed up the drug development process, to advising on the design of clinical trials and advising on what outcomes are meaningful for patients when drugs are being developed. We work at UK level with the Medicines and Healthcare Products Regulatory Agency (MHRA), and both of our founders are members of the MHRA’s Patient Consultative Forum.
Duchenne UK and the DMD Hub are launching a new pilot project aiming to provide fair and equal access to DMD trials across the UK
In a new transatlantic collaboration, Duchenne UK and the DMD Hub, PPMD and RTI International are conducting a study to explore attitudes towards gene therapy and gene editing from adults with Duchenne muscular dystrophy (DMD), parents and caregivers of children with DMD, and clinicians.
The Clinical Trials Lectureship grant enabled Dr Michela Guglieri to act as the Clinical Research Team Leader within the John Walton Muscular Dystrophy Research Centre in Newcastle
In the last few months, the DMD Hub has recruited two new staff members to work with Emma Heslop, the DMD Hub Manager, and to look at new areas where the DMD Hub can develop support.
The European Medicines Agency (EMA) has approved the first measure collected using a digital wearable device for use in clinical trials for Duchenne muscular dystrophy.
Duchenne UK and Project HERCULES announce the launch of a new patient centred, quality of life tool designed to support faster access to new treatments for people living with Duchenne muscular dystrophy
We're funding more posts as part of the DMD Hub so that more boys are now able to take part in trials for potentially life-changing treatments.
Selecting which DMD treatments to advance to human testing is a critical decision. In 2020, Duchenne UK joined with US-based charity Charley’s Fund to improve the data collected in preclinical research.
Dr Emily Whitehouse is the latest consultant to join the fight against DMD at the Royal Manchester Children’s Hospital, which is part of the DMD Hub network of clinical trial sites.
We set up HERCULES, a collaborative project to increase the chances of DMD patients accessing treatments.
We founded the DMD Hub to increase the number of UK sites able to run clinical trials for DMD.
Find out about the research we fund to find the most effective treatments for DMD.
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