Research strategy
Innovation in medical research is core to our strategy. Our priorities are informed by people with Duchenne muscular dystrophy and their parents and carers, ensuring that we are sharply focused on the research innovations that will create the biggest impact for them.
About our strategy
Our focus today is built on our successes to date, as a funder, innovator and enabler of positive change for our community. Our intention is to continue to fund, drive and enable the most effective research to develop new treatments and the best clinical care, and ensure that people with DMD can access them as quickly as possible.
We collaborate with drug developers in industry and academia, with the clinical community, and with other patient organisations and key stakeholders nationally and internationally, to ensure that as a funder and a partner we bring the most value and make the biggest impact for people with Duchenne.
We are smart in how we fund research and drive its commercialisation to ensure the fairest return of investment to us, so that we can invest more funding into research to transform lives.
Our strategic priorities
Underpinned by our flagship programmes (Duchenne Hub UK, Duchenne Care UK, HERCULES and Duchenne UK Connect), our strategic research priorities are:
Accelerate the development of new treatments through funding of translational and early-stage clinical programmes in industry and academia
Drive the highest standards in Duchenne clinical care through research generating evidence to improve clinical practice.
Support clinical research infrastructure to deliver clinical trials in the UK and accelerate access to approved treatments through our flagship programmes
Short-term research goals
Therapeutics
Significant progress has been made in recent years leading to a healthy pipeline of new therapeutic approaches for Duchenne in preclinical and clinical development. These include second generation dystrophin restoration approaches, new approaches to cell and gene therapy, and novel anti-inflammatory and antifibrotic strategies. Alongside novel therapies, opportunities also exist to investigate known drugs that are not already used in Duchenne, such as drugs that could address cardiac and psychological involvement. We will continue to support and encourage this progress, to accelerate the development of effective treatments for all people living with Duchenne.
Care
We will also support the continued improvement and harmonisation of care for Duchenne patients, ensuring Duchenne patients receive the best care available. This includes investigating the best models for delivering existing care, as well as the assessment of new care strategies and new non-drug treatments to improve the lives of people living with Duchenne. We will accelerate the validation and translation in clinic of these approaches, working closely with our Duchenne Care UK programme.
Clinical Research Infrastructure
We will also continue to invest in the clinical research infrastructure through our Duchenne Hub UK programme, to ensure the right resources are in place to deliver trials and newly approved treatments to patients.
Longer term goals
Over the coming years, we will continue to partner with clinicians, researchers, industry and others in the community, to drive forward innovative research for a cure - this will include driving genetic approaches that can treat the muscles, the heart and the brain, as well as continuing to support research into technologies and therapies that allows people living with Duchenne to live life to the fullest. We will continue to bring the voice of people with Duchenne into the research agenda, setting priorities that reflect their priorities.
