We are the UK’s leanest Duchenne research charity. In the last 5 years (as Duchenne Children's Trust and Joining Jack) Duchenne UK has raised over £10 million

We've spent £6.5m to date and committed a further £3.5m to accelerating research.

Please read our Research Pipeline to see where and how we can add value to the drug development pathway, and how we can help to accelerate research that will benefit the whole community.

To do this we are engaging at each stage of the drug development pathway.

We have achieved some incredible results, with the help of donors, partner charities and researchers:

  • Duchenne UK partnered with Duchenne Research Fund and Joining Jack in 2014, to commit $5million to help fund preclinical work for Solid Biosciences gene therapy compound. Just three years later, that compound is NOW IN THE CLINIC being tested on boys with Duchenne!
  • Co-funded a successful Phase I trial for a steroid alternative trial of Vamorolone, previously known as VBP15. As a result of our investment, the company was able to raise a further $12million for the Phase II clinical trial which is NOW UNDERWAY!
  • Helped to advance and accelerate a clinical trial to test the safety and efficacy of Tamoxifen as a treatment for DMD, by funding a project manager and clinical trial sites for the study. We sought regulatory guidance with the tamoxifen team, at the European Medicines Agency (EMA).
  • Led a collaboration of 6 companies developing treatments for DMD, to launch Project HERCULES, a unique, innovative and groundbreaking multi-stakeholder collaboration led by Duchenne UK to help them better and more efficiently engage with NICE and other global bodies who decide how government should pay for medicines.
  • Set up the DMD Hub, a network of clinical trial sites to expand access for patients wanting to join clinical trials. Duchenne UK is using an innovative tapered funding model to pay for staff in selected sites to help deliver clinical trials. The Hub will educate patients about trials coming up and will be a point of contact for companies wanting to set up studies quickly and efficiently.
  • Funded Crispr/Cas9 gene editing to fix a faulty dystrophin gene in DMD cells from a patient with a duplication and deletion.
  • Hosted free patient information days to educate and inform patients about clinical trials and what opportunities there are coming up in the UK.
  • Sped up access to promising drugs through lobbying for changes to the regulatory framework through the Early Access to Medicine’s scheme. Our founder was praised in the House of Commons for the Charity’s work in lobbying to accelerate the time it takes for drugs with urgent unmet medical needs to be approved.
  • Lobbied for early approval of promising drugs: Emily and Alex have given evidence in parliament and to the Food and Drug Administration (FDA).
  • Spent over 90p in every £1 raised directly on research grants by being the most efficient, transparent and volunteer-supported organisation working on a cure for Duchenne.
  • Made powerful links across the Duchenne Community: parents, scientists, clinicians and governments to identify and fund the best treatments worldwide for Duchenne through our leadership and sponsorship of Treat NMD, a global network of scientists and clinicians.
  • Raised the profile of Duchenne in the UK both through media interviews on television, radio and in newspapers, and through the support of the world of rugby and Owen Farrell’s JJ salute.

Our Impact In Figures:

  • £6.5m spent on accelerating research and a further £3.5m committed.
  • $42.5m of private investment in Duchenne Biotech firm Solid GT as a result of our joint seed investment with 3 other charities.
  • $12million of government funding for Phase II study of VBP15 as a result of our initial co-funding with Muscular Dystrophy America and The Duchenne Research Fund.

Duchenne UK is funding 50 research projects including 3 clinical trials.

5 additional trials now running as a result of one clinician we are co-funding.

16 clinical trial staff in the UK co-funded by us.

Since we established the DMD Hub, Duchenne UK have committed an additional £400k to fund clinical Hub posts which include:

  • Clinical research nurse at Alder Hey Children’s Hospital
  • Clinical research nurse at Newcastle Hospital
  • Posts for new Hub sites at Leeds, Glasgow and Bristol