04:48pm Monday 14 October 2019

Scientists to develop new treatment for Duchenne muscular dystrophy


The team of researchers from several European countries and the USA, called the MDEX Consortium, is led by UCL scientist Professor Francesco Muntoni, Institute of Child Health, and includes Professor George Dickson, from Royal Holloway’s School of Biological Sciences.

DMD is a devastating and life-limiting condition that affects one in about 3,500 male births in the general population, with about 100 new cases diagnosed in the UK each year. The condition leads to progressive muscle weakness due to the breakdown and loss of muscle cells.

Patients lack a single important protein in their muscle fibres called dystrophin. By the age eight to 12, boys become unable to walk and by their late teens or early twenties the condition can become severe enough to limit life expectancy.

The EU grant totalling 5.5 million euros comes after a MDEX Lancet paper in July, 2011, announced that a drug known as an ‘antisense oligonucleotide’, could be used safely to restore dystrophin production in a subset of DMD boys, by “skipping” one exon. However, different antisense oligonucleotides are required to skip different exons and so a panel of such drugs is required to ensure as many boys as possible can be treated.

In theory, this approach could work for at least 70% of DMD sufferers. However, further research to test this is required first.

Professor Dickson said: “Royal Holloway’s role is to lead a team effort to design and optimise the new therapeutic exon skipping drug, and we are very excited to contribute our expertise, and be involved with this excellent consortium of clinicians, scientists and industry partners who will perform these clinical trials in DMD patients.”

Royal Holloway will have a budget of 200,000 euros (approximately £160,000) for its research.  

Sarepta Therapeutics has made the work possible by not only providing the drug, but also half of the drug production and clinical trial costs.

Royal Holloway, University of London home

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