Pioneering research into Duchenne muscular dystrophy secures commercial funding

02-Apr-2013 - United Kingdom

Royal Holloway is giving money generated by its research into Duchenne muscular dystrophy to the Muscular Dystrophy Campaign. Families affected by the condition are also visiting laboratories on campus to find out from the scientists about new drug developments and see the work being carried out.

Affecting just 1 in 3000 young boys, Duchenne muscular dystrophy is a life-shortening condition, which causes muscles to weaken and waste over time leading to increasingly severe disability.

Professor George Dickson from the School of Biological Sciences at Royal Holloway has been leading a team of scientists looking into pioneering treatments, including exon skipping, a process that looks to encourage cellular machinery to 'skip over' an exon which makes up part of the gene. It is thought that by skipping one or two exons, it may be possible to treat around 83% of the genetic errors causing Duchenne muscular dystrophy.

A pharmaceutical company is now looking to collaborate with the team to develop drugs that could be used for treating the condition and has paid a fee to secure rights to the research.

Professor George Dickson said : "We are delighted to be able to return money to the Muscular Dystrophy Campaign who originally funded this research in the early stages.

"The revenue generated shows that our work is having a real impact. Our research is directly leading to new treatments and hopefully drugs to treat this condition which can be crippling for those affected by it."

Dr Marita Pohlschmidt, Director of Research at the Muscular Dystrophy Campaign said: "We are at a crucial stage in research into finding treatments for Duchenne muscular dystrophy. A therapy that will change the lives of children with Duchenne is almost certainly on the horizon. However, this is a very complex genetic condition, and exon-skipping will not work for all those affected. The additional funding we will receive from Professor Dickson's work can be ploughed back into our other lines of research, and will help us ensure that every child with Duchenne will one day benefit from treatment."

Neil and Karen Robinson, whose five-year-old son Thomas has Duchenne muscular dystrophy, will take part in the laboratory tour. They are raising funds for research into Duchenne muscular dystrophy through the Muscular Dystrophy Campaign's Duchenne Research Breakthrough Fund.

Karen said: "It's a really great thing for us as a family to be able to physically see where the money we have been raising goes. So often people just send off funds raised by charity events and challenges, and trust that it is put into good work. Visiting the lab gives us the opportunity not only to meet the people who are carrying out the research we back, but also to explain to family, friends and all those who support us exactly where their donations go."

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