Funding for breakthrough in MD research
There is a bit of optimism on the horizon for children who have Muscular Dystrophy. Following a briefing last year, Hon Nick Goiran MLC and I met with Health Minister Kim Hames to tell him about a revolutionary breakthrough in research into a type of MD called Duchenne which affects up to three boys each year in Perth, and one in every 3500 boys worldwide.
A genetic error means that a particular protein, which acts like a molecular shock absorber and strengthens and stabilises muscle fibres, is not made properly. As a result, muscles weaken to the point where walking, and eventually breathing, is not possible. In a process called exon skipping, the researchers use molecular scalpels to reprogram the body’s machinery and snip out the disease-causing part of the gene message - similar to the way a patch is used to remedy a computer software glitch.
Mice trials have shown dramatic effects, and it is now time to undertake clinical trials on humans.
I am delighted that, as a result of our discussions, Minister Hames has announced funding of $647,000 over three years for these clinical trials, which could also have implications for other conditions such as cancer, asthma, Alzheimer’s and diabetes.
Watch Lesley and Conor tell their story, and take a moment to learn how an NDIS could change their lives.
A genetic error means that a particular protein, which acts like a molecular shock absorber and strengthens and stabilises muscle fibres, is not made properly. As a result, muscles weaken to the point where walking, and eventually breathing, is not possible. In a process called exon skipping, the researchers use molecular scalpels to reprogram the body’s machinery and snip out the disease-causing part of the gene message - similar to the way a patch is used to remedy a computer software glitch.
Mice trials have shown dramatic effects, and it is now time to undertake clinical trials on humans.
I am delighted that, as a result of our discussions, Minister Hames has announced funding of $647,000 over three years for these clinical trials, which could also have implications for other conditions such as cancer, asthma, Alzheimer’s and diabetes.
Watch Lesley and Conor tell their story, and take a moment to learn how an NDIS could change their lives.
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Ph: +61 (08) 9923 9211; Fax: +61 (08) 9923 9222; Email: brian.ellis@mp.wa.gov.au
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