Children affected by Duchenne Muscular Dystrophy march to Downing Street

Children and their families who are affected by Duchenne Muscular Dystrophy will be marching to Downing Street and the Department of Health today.

The are demanding an end to the under-funding for their medical care and research for the severe muscle wasting disease that has no cure.

It affects boys and young men and incredibly rarely girls too.

Those who develop the disease usually end up in a wheelchair by the age of ten and without treatment, die in their late teens or early twenties.

Katherine Wedell’s son Isaac has Duchenne. Explaining more about his condition she said: “Duchenne Muscular Dystrophy is a devastating degenerative condition and my six year old son Isaac has it. There is currently no cure. Isaac, who is currently dancing and swimming and riding his trike to school, faces a wheelchair by age ten and death in his teens or twenties.”

Wanting to save her son and other boys affected by the conditions, she is calling for the Government to make sure there are specialist care teams within fifty miles of a family’s home, ending the post-code lottery of care.

She is also asking for more funds to be made available for research as for the first time, a generation of children could find their wish for a cure become a reality.

Neuromuscular experts are also calling for the Government to increase funding for research and clinical trials for treatments to give sufferers a chance of living.

Duchenne currently affects around 2,500 people in the UK and about 40,000 worldwide.

Though the gene that leads to the disease was discovered in 1986, there has only been £2.2 million of funding allocated since then.

Recent clinical trials into the effectiveness of Exon Skipping as a gene therapy is showing promising results.
 
Scientists say that a cure is incredibly close but more funding is needed.

Professor Dame Kay Davies at the University of Oxford said: “We are now close to finding an effective treatment or cure for Duchenne patients, but we need the funding to continue so that we can ensure that the basic science advances can be delivered to the clinic. I sincerely hope that Action Duchenne are successful in their call for funding to find a cure for this devastating condition.”

Parents Sharon and Chris Scutt are desperate for a treatment or cure for their son Tom, who has Duchenne.

They said: “It is urgent and of vital importance that the government provide the funding needed for research into DMD so that new drugs and treatments, currently in clinical trials, can be made available as quickly as possible. Any delay will cause loss of life.
 
“Specialist centres must be available, within reasonable access, for all DMD patients so that new treatments can be quickly and safely delivered. Up to date news on current research which gives us hope for our son’s future with support from charity and other families.”

For more information, visit: www.mdex.org.uk