What We Do Our Strategy To End Duchenne Our Mission Duchenne UK is run by parents, fighting every day to speed up treatments for all our children and those living with DMD. Our sons have taken part in clinical trials. We understand the pain and the challenges families face. And we are fighting for all of us. We can learn from our experiences on the front line, because we live with Duchenne every minute of every day. And we can act. Read about what we did here to address clinical trial capacity in the UK. Patients with DMD are at the heart of everything we do. Our approach is 100% focused on advancing research – and getting treatments into the clinic and to patients as quickly as possible. We actively seek out projects and possible therapies that could benefit this generation of patients. We invest globally in both basic science and translational research. Our Scientific Advisory Board comprises scientists, doctors and drug developers so that we look at each project and ask: Is this good science? Can this be taken into the clinic? What hurdles exist? Is this being replicated anywhere else? The Multiplier Effect Duchenne UK has a pioneering and ambitious research approach, which means that the money you raise for us can go further. On certain projects, we create a 'multiplier effect'. Duchenne UK provides research spend to prove that a drug is ready for trial, which is then a catalyst for additional investment. By bridging the notorious valley of death in drug development, we are striving to bring promising therapies out of the laboratory and into the clinic where they can be tested on patients. Along with the Duchenne Research Fund, we invested $5million in a gene therapy project for Solid GT. That seed funding enabled them to raise $42m of private investment to fully fund further clinical trials. We invested $1million of seed funding to co-fund the Phase I study of Vamorolone (VBP15). Following the trial's success, the company was able to raise $12m of government funding for the Phase II study of Vamorolone (VBP15). Our Integrated Approach Our strategy has five elements to it, all of which are integral to accelerating and advancing research. Funding the research is the first crucial step. But on its own it isn’t enough. We need to look at the overall landscape of research and drug development, and our strategy has been developed to reflect this. 1: Fund and accelerate research and early phase clinical trials Duchenne UK has a pioneering and ambitious research approach. We fund a variety of research, but we focus on translational projects, using our funds to help get promising therapies out of the laboratory and into the clinic where they can help patients. We actively seek out projects and possible therapies that could benefit this generation of patients. We invest globally in basic science and translational research. Here are the projects we are currently funding, but we can and will consider all aspects of research. 2: Facilitate and accelerate research We are committed to accelerating the pace of research, ensuring that we remove potential obstacles and roadblocks that lie in the path of drug development. It is vital that the research we fund has the potential to translate into clinical trials, and so we fund the doctors, nurses and physiotherapists necessary to conduct trials in the UK. We also support and encourage national and international collaboration to allow researchers and clinicians to collaborate and progress research. We are proud to support the Treat-NMD network and their bi-annual TACT meetings to review potential clinical trials for Duchenne muscular dystrophy. 3: Work alongside industry We work with industry, advising on clinical trial designs. We have advised on the protocols of a number of different trials. 4: Continued engagement with regulators and policy makers Duchenne UK engages with policy makers and regulators on many issues, from campaigning for Early Access to Medicines to engaging with the Health Technology Appraisal system. We are involved in advising regulators on the government’s Accelerated Access Review and we advise HTAs on the design of clinical trials and what outcomes are meaningful for patients when drugs are being developed. 5: Collaborate with families and charities globally Duchenne UK has strong links with families in the UK and globally. Families fundraise for us and we have a Patient Advisory Board to give lay reviews of the types of projects we are considering supporting. We collaborate globally, and our co-founder Alex Johnson sits on the board of UPPMD, United Parent Project Muscular Dystrophy.