Duchenne UK funds new technique for finding effective DMD treatments
Duchenne UK is pleased to announce a new grant to support the search for new Duchenne muscular dystrophy (DMD) treatments using cutting-edge technologies.
Together with our charity partner Joining Jack, Duchenne UK will grant £65,376 to Professor Jordi Diaz Manera of Newcastle University for a three-year research project to investigate drugs that could delay muscle degeneration.
Professor Diaz Manera and his team have developed a new molecular biology technique which enables them to look inside muscle cells from DMD patients. This will help them to understand which treatments are most effective in preventing fibrosis, a process which happens when muscle cells die and are replaced by fatty tissue.
Fibrosis can lead to failure of the heart and respiratory muscles, which is the most common cause of death in adults with DMD.
Professor Diaz Manera and his team have already analysed nearly 200 potential drugs and identified ten promising candidates for further investigation. The researchers will combine this drug screening approach with examining the DMD muscle cells. Combining these two approaches could provide the evidence needed to take promising anti-fibrotic medicines into clinical trials.
While gene therapies could be transformative for people living with DMD, the progressive nature of the disease limits their impact. Combining gene therapies with drugs that could reduce muscle deterioration, such as anti-fibrotics, could help to maximise the effect of these treatments. Anti-fibrotics could also be used to treat patients who are not eligible for gene therapy, and for those in the later stages of the disease.
Professor Diaz Manera said:
“We are very pleased to be able to establish this collaboration with Duchenne UK to identify new drugs that can reduce or stop the progression of fibrosis and fatty infiltration in the muscles of patients with this terrible disease. We are confident that this is a therapeutic approach that can provide beneficial results for patients and that will allow us to advance research into DMD.”
Emily Reuben and Alex Johnson, Founders of Duchenne UK said:
“Professor Diaz Manera has developed an exciting way to discover new treatments to effectively treat fibrosis, and we are pleased to be helping his team look for transformative treatments for DMD patients of all ages.”
What is Duchenne muscular dystrophy?
Duchenne muscular dystrophy (DMD) is a genetic disease diagnosed in childhood; it is the most common and severe form of muscular dystrophy. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and a shortened life expectancy. It almost exclusively affects boys. In the UK there are around 2,500 boys affected and around 300,000 worldwide.
Who is Duchenne UK?
Duchenne UK has one clear aim – to end Duchenne, a devastating muscle-wasting disease. As the leading DMD charity in the UK, we connect the best researchers with industry, the NHS and families to accelerate every stage of drug development. Together, we will find effective treatments for this generation of people living with DMD.
Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.