Searching for a new treatment
Each year in the UK, around 100 boys are born with Duchenne muscular dystrophy, a rare and life-limiting genetic disease.
The disease is caused by a faulty gene that doesn’t make enough – or any – of the protein dystrophin. The lack of this protein causes muscle damage and progressive weakness, often first noticed in early childhood and will gradually worsen overtime.
Sadly, there is no cure – and although some treatments are available to help slow its progression, they do not benefit all affected children.
That's why we're funding research, led by Professor Angela Russell at the University of Oxford that hopes to identify a drug compound that could become an effective new treatment for all children with Duchenne.
Professor Russell and her team are exploring a number of new compounds and whether they can boost utrophin levels, a protein that is found naturally in muscles in small amounts, that has the potential to act as a substitute for dystrophin.
If Professor Russell’s research proves successful, it could result in a new drug treatment that could help improve the lives of all children with DMD.
Our goal is to identify a drug compound that has the potential to become an effective new treatment for ALL children with Duchenne
Our charity partner, LifeArc, has kindly pledged to fund half of this project cost. At Action, we are now fundraising for the remaining costs to help drive this research forward.