A new treatment for Duchenne muscular dystrophy could be available within five years.
Professor Kay Davies, an Oxford University researcher, has said trials of a drug to increase levels of the protein utrophin, to maintain the muscles, would start later this year and could help sufferers worldwide.
“Duchenne is horrible for these boys. They normally get diagnosed at the age of four or five, and they suffer from a progressive muscle wastage which leaves them in a wheelchair by the age of 12,” she said. “Somewhere in the next five years, we will be able to do something for these boys, to stop them from going into a wheelchair, and perhaps prolong their life and improve their quality of life.”
Oxford mother and Action Duchenne advocate, Kathy Wedell, whose 12-year-old son Isaac has Duchenne muscular dystrophy, said taking part in the trials “fills her with hope” for all sufferers.
“The diagnosis when Isaac was four left us devastated. DMD means losing the ability to move and dying in your 20s or 30s. As a parent it means seeing your child not being able to do the things he loved. It’s wonderful to be part of a trial, not just for Isaac, because it will provide the evidence for a treatment for others who will benefit in the future.”
Trials in mice found the increased levels of utrophin could compensate for a lack of a similar protein called dystrophin, which people with Duchenne muscular dystrophy lack.
Professor Davies, from the university’s department of physiology, anatomy and genetics, has been working on finding a treatment for more than 20 years. She said: “It affects every population in the world and we need to have a treatment in pill form. This is why I am so keen on our approach, because this is something anyone can take with their breakfast or dinner.”
For further information please contact our CEO and Head of Research Diana Ribeirodiana@actionduchenne.org