Duchenne muscular dystrophy: developing a badly needed new treatment for boys with this devastating condition
This research was completed on 31 August 2016
Published on 5 February 2014
Duchenne muscular dystrophy is a devastating condition that’s diagnosed in around 100 boys every year in the UK – typically when they are just three to five years old.1,2 Boys with the condition experience a relentless deterioration of their physical abilities, eventually becoming heavily dependent on others for round-the-clock care. Sadly, there is no cure and the condition is fatal. Researchers led by Professor Volker Straub, of Newcastle University, are in the laboratory stages of testing a much-needed new treatment – with the ultimate hope of improving boys’ lives.
How are children’s lives affected now?
“Boys with Duchenne muscular dystrophy experience progressive muscle weakness and wasting,” explains Professor Straub. “By around 10 to 14 years of age, they start needing to use a wheelchair. Dependence on others increases with time as symptoms get gradually worse.”
Teenage boys and young men with the disease often need help with everyday tasks such as eating and washing, and a ventilator to help them breathe at night.
“Good care can have a profound impact on both quality of life and life expectancy,” says Professor Straub. “Many boys learn to live with their condition and maintain a positive outlook.”
However, the disease remains fatal, with many of those affected losing their lives by their late twenties.
“Understandably, parents are normally devastated to see their child gradually deteriorating with this terminal, incurable condition,” says Professor Straub. “Better treatments that improve boys’ quality of life and slow the progression of their disease are urgently needed.”
How could this research help?
Professor Straub and his team are developing a new treatment. “We’ve known for many years that boys with Duchenne muscular dystrophy have too much calcium in their muscles,” he explains. “We’ve identified a possible new medicine that might reduce this calcium overload and we are testing it in the laboratory.”
“As we are still in the laboratory stages of our work, we don’t know for sure yet whether or not the new treatment will work” continues Professor Straub. “However, our vision is to develop a medicine that increases muscle strength and slows down disease progression – helping boys with Duchenne muscular dystrophy to walk longer without a wheelchair and improving their overall quality of life.”
Around one in every 3,500 boys in the UK has Duchenne muscular dystrophy.3 Through this exciting research, Professor Straub and his team are determined to do everything they can to help.
|Project Leader||Professor Volker Straub MD PhD FRCPCH|
|Location||The MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University|
|Grant awarded||18 November 2013|
|Start date||1 March 2014|
|End date||31 August 2016|
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1. Muscular dystrophy campaign. Duchenne muscular dystrophy. http://www.muscular-dystrophy.org/about_muscular_dystrophy/conditions/97... Website accessed 13 January 2014.
2. National Institute of Neurological Disorders and Stroke. NINDS Muscular Dystrophy Information Page. http://www.ninds.nih.gov/disorders/md/md.htm Website accessed 27 January 2014.
3. NHS Choices. Muscular dystrophy. http://www.nhs.uk/conditions/Muscular-dystrophy/Pages/Introduction.aspx Website accessed 13 January 2014.