Drug trials for muscular dystrophy sufferers to get underway

Young boys and men living with Duchenne Muscular Dystrophy could have access to a new treatment within the next five years, a leading expert has said.

Speaking at the Oxfordshire Science Festival, Professor Kay Davies said that trials of a new drug, designed to increase the levels of the protein utrophin in sufferers, were set to get underway towards the end of 2016.

If approved for wider use, the course of treatment could make a considerable difference to those living with the disease.

Prof Davies, who works at Oxford University’s department of physiology, anatomy and genetics, has spent the past two decades trying to find a more effective drug to help sufferers.

Trials in mice have already yielded very encouraging results and the treatment is unusual in that it can be made available in pill-form.

Prof Davies said: “Duchenne is horrible for these boys. They normally get diagnosed at the age of four of five, and they suffer from a progressive muscle wastage which leaves them in a wheelchair by the age of 12.

“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.”

Twelve-year-old Isaac Wedell will be among those taking part in the trial and his mum Cathy said that the research offered real hope to the thousands living with the condition.

“The diagnosis when Isaac was four left us devastated,” she told the BBC.

“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.”

At EnViva Paediatric Care, our experienced staff can provide complex nurse-managed homecare for babies, children and young people with conditions including muscular dystrophy. For more information about the services we provide, please contact us today.