Our current research projects
We are committed to funding research that makes a real difference for people living with Duchenne.
Our projects range from cutting-edge genetic therapies topractical studies that improve everyday care.
Drugs to address unmet needs
Duchenne UK have a successful history of supporting drugs, such as vamorolone, through research and development all the way to approved treatments. We continue to support the development of drugs that can have an impact on unmet needs for those living with Duchenne.
Through our cardiac-focussed funding call, for example, we are funding Great Ormond Street Hospital to conduct a clinical trial in Duchenne patients for an existing heart disease drug, to understand whether this drug may be effective in improving Duchenne-associated cardiomyopathy.
Gene and cell therapies
Duchenne UK has a strong history of funding gene and cell therapies for Duchenne, having been an early supporter of Solid Bio’s gene therapy programmes, among others. Gene and cell therapies are a rich area of ongoing research for Duchenne, and Duchenne UK continue to support several active programmes in this therapy area.
Examples include our recent funding for MyoGene Bio, a San Diego-based biotech company, to progress pre-clinical development of an innovative gene editing therapy for Duchenne. The cutting-edge CRISPR/Cas9-based therapy, MyoDys45-55, aims to permanently delete a mutation hotspot in the Duchenne gene. The approach is designed to restore the reading frame and produce a shorter but functional dystrophin protein, similar to that found in individuals with Becker muscular dystrophy.
Further examples include our ongoing funding for a clinical trial at the University of Minnesota Medical School to evaluate muscle progenitor cells (a biological cell that can change into a specific cell type) as a possible treatment for Duchenne.
Non-drug treatments
Duchenne UK recognises the importance of clinical care and non-drug treatments for Duchenne, not only to improve quality of life, but to drive better outcomes for patients. This research work sits alongside the Duchenne Care UK programme, aiming to advance care as well as setting consistent standards for its delivery.
Examples of current work include our funding of a clinical trial for hydrotherapy in boys with DMD at Royal Preston Hospital and Manchester Metropolitan University and a nutrition study at the University of Glasgow to understand the impact of factors like protein intake for muscle maintenance, vitamin D and calcium for bone health, and the role of anti-inflammatory diets. We also take an interest in the application of digital technologies to improving care delivery, funding external work in this area, alongside internal development efforts, such as our Duchenne UK Connect platform.
Building research capacity
Effective research infrastructure and expertise is vital to the delivery of good care and new treatments for Duchenne patients in the UK.
Duchenne UK have a strong pedigree of funding UK research capacity, such as Duchenne Hub UK, as well as fellowships and PhD studentships for the next generation of scientists dedicated to neuromuscular research. Current examples include our ongoing support for clinical trials infrastructure at the John Walton Muscular Dystrophy Research Centre in Newcastle and funding to support neuromuscular services at Leeds Teaching Hospital.
