Our Impact We are the UK’s leanest Duchenne research charity. In the last 3 years (as Duchenne Children's Trust and Joining Jack) we have raised over £8.6 million. 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. Collaborated with UK patient groups to raise £1.2 million to fund 16 critically needed clinical posts at leading hospitals in the UK to boost trial capacity and run clinicial trials. Co-funded a doctor who is now running 7 clinical trials, and one natural history study at Newcastle. 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: Duchenne UK is running 50 research projects including 3 clinical trials. 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.