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UWA researchers have been awarded more than $1.2 million in US funding for research into the treatment of the devastating muscle-wasting disease Duchenne Muscular Dystrophy (DMD).

Molecular biologist Steve Wilton, who will be a keynote speaker at this week's Australasian Gene Therapy Society meeting in Sydney, and his UWA colleague Sue Fletcher received the funding through the National Institutes of Health (NIH).

"NIH funding is extremely competitive, especially for research based outside the US. We are very appreciative that our research has been recognised and deemed worthy of such significant funding," Professor Wilton said.

DMD is a relentlessly progressive and incurable muscle wasting disorder, and one of the most common serious genetic disorders to affect children around the world. Each year, at least three boys born in Perth will have the disease.

Professor Dame Kay Davies, a world expert on DMD and Professor of Anatomy at the University of Oxford, has described Professor Wilton's research as "the most important work into a treatment for DMD in the world at the moment".

The NIH grant, which includes more than $314,000 per year over four years, follows UWA's signing of an exclusive worldwide licence agreement with US-based bio-pharmaceutical company AVI BioPharma in November last year to a patent related to the treatment of DMD.

According to its website, the NIH looks for unique projects of high scientific calibre that are relevant to public health needs. NIH funds support the advancement of fundamental knowledge about the nature and behaviour of living systems, to extend a healthy life, and reducing the burdens of illness and disability.

Professor Wilton, Head of the Molecular Genetic Therapies Group at the UWA Centre for Neuromuscular and Neurological Disorders, said clinical trials in the UK using a compound developed in Perth for the treatment of DMD had yielded promising results.

"Now, for the first time we feel as though we may be able to give hope to some of those families who have been affected by this terrible disease," he said. "While it is unlikely that we can reverse the effects of the disease, we believe that antisense treatment may reduce the progression of the disease, improve muscle function and quality of life."

Professors Wilton and Fletcher's work on the innovative use of antisense technology to restore dystrophin expression in Duchenne muscular dystrophy was showcased in the December 5, 2008 edition of the prestigious international journal Science .

The Muscular Dystrophy Association of WA provides a support network for West Australian families affected by muscular dystrophy. It also funds research such as Professor Wilton's, at the world-leading Australian Neuromuscular Research Institute.

About Duchenne Muscular Dystrophy (DMD):

Approximately one in every 3,500 boys worldwide is afflicted with Duchenne muscular dystrophy, with one third of cases presenting with no prior family history of disease. DMD is a devastating and incurable muscle-wasting disease associated with specific errors in the gene that encodes dystrophin, a protein that plays a key structural role in muscle fibre function and stability.

Symptoms usually appear in boys before the age of six years. At this age, affected boys have difficulty in keeping up with their peers, may appear clumsy and fall easily. By age 10, boys have difficulty walking, and are confined to a wheelchair by age 12. Eventually, all muscles are affected and patients experience increased difficulty in breathing.

The muscle wasting is relentlessly progressive and death usually occurs before the age of 25. The outpatient cost of care for a non-ambulatory DMD boy is among the highest of any disease. There is currently no effective treatment for DMD, but for the first time in decades, there are a range of promising therapies under development.

Media references

Professor Steve Wilton (+61 8) 9346 3967 / (+61 4) 17 982 365
(UWA Centre for Neuromuscular and Neurological disorders)
Janine MacDonald (UWA Public Affairs) (+61 8) 6488 5563 / (+61 4) 32 637 716