Our Impact (New)


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

  • Proved that a new gene therapy approach works in animal models. Our investment of $5million allowed the US biotech running the project to raise a further $42million to fund the Phase I/II.
  • 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 starting in the US this month, and in the UK in December.
  • Funded Crispr/Cas9 gene editing to fix a faulty dystrophin gene in DMD cells from a patient with a duplication.
  • Set up the DMD HUB to make more clinical sites trial ready by providing training, mentoring and resources to help them achieve the requirements set by industry to run clinical trials. Duchenne UK’s mission for the DMD Hub is to ensure that all patients with DMD, including children and adults, have the opportunity to access clinical research opportunities.
  • 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:

  • 3 clinical trials, 13 research projects
  • 8 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.
  • £4m spent on direct research grants. 
  • $42m 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.

Published on 10 May 2017

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