10 June 2010
Action Duchenne lobbies Government for care levels that will extend Duchenne childrens’ lives by up to 15 years, in face of threatened cut backs
Parents and boys living with Duchenne march on Downing Street to raise awareness of heartbreaking disease and demand an increase in investment to end to Postcode Lottery for medical care
On 16 June, boys living with Duchenne Muscular Dystrophy, their families and supporters will be lobbying MPs and marching on No. 10 Downing Street and the Department of Health to demand an end to the continued serious under-funding for medical care and research into the condition. In January and February 2010 internationally agreed Standards of Care for Duchenne Muscular Dystrophy were published in the Lancet Neurology. The Duchenne families are calling on the Government to ensure that the levels of care for these vulnerable children are not cut and that recommended ‘Best Practice’ care is available to all children and young men with Duchenne. It has been proven that such care adds 10-15 years life expectancy and considerably improves quality of life. People living with Duchenne will be travelling to London to take part in the lobby from all around the country. For many this will be a difficult journey due to the severe nature of the condition.
The lobby action is being organised by Action Duchenne, the only UK charity dedicated solely to raising awareness and raising funds for research into treatments and finding a cure, and providing support for families living with Duchenne.
The families will also be calling for funding for medical research to find a cure for Duchenne. Recent clinical trials into the effectiveness of Exon Skipping as a gene therapy for the treatment of Duchenne have shown promising results and scientists say that a cure is tantalisingly close, but more funding is needed.
The group will meet at Central Hall, Storeys Gate, Westminster to register and sign Open Letters. At 11am they will march together to Downing Street and the Department of Health. From 12 noon until 2pm families will lobby their MPs inviting them to attend a meeting at 2pm. The meeting will include presentations from Dave Anderson, MP and Chair of the All Party Parliamentary Group for Muscular Dystrophy, Dr. Karen Rafferty, Nick Catlin, CEO of Action Duchenne and Dr, Matthew Wood.
Duchenne Muscular Dystrophy currently has no cure. It is a severe muscle wasting disease that affects boys and young men, and very occasionally girls too, resulting in children being in a wheelchair by aged 10 and without treatment, dying in their late teens or early twenties. Duchenne affects 2,500 people in the UK and about 40,000 people worldwide. The gene that causes Duchenne was discovered in 1986, and yet since then the Government has provided only £2.2m of funding for research and clinical trials for treatments and a cure.
Access to the type of multidisciplinary care described in the Standards of Care document could add as much as 10 to 15 years to life expectancy and also improve quality of life. Young men that receive the full spectrum of care are expected to live until their late twenties and into their thirties, Denmark being a good example of this with young men often living into their 30s and 40s. Action Duchenne is lobbying the UK government to provide a similar standard of care to boys and young men in the UK.
Action Duchenne will be calling on the Government to:
• Set up a network of Centres of Excellence in the UK for Duchenne patients that provide the multi disciplinary medical care recommended in the Standards of Care for Duchenne published in the Lancet Neurology Oncology in March 2010.
• The Centres of Excellence should be centrally funded by the new proposed National Commissioning Framework
• Key services for personal care, transport, housing, further education, vocational training for those living with Duchenne must be in place and properly coordinated at the transition stage to adult provision.
• £30m of ring fenced funding should be allocated to the Centres of Excellence to develop them as clinical trial sites for the research and development of new drugs.
• The Government should promote funding partnerships directly with the Biotech industry to stimulate further research to Cure Duchenne Muscular Dystrophy.
Nick Catlin, CEO of Action Duchenne said, “The article published in Lancet Neurology in January and February this year detailed the required care standards for patients with Duchenne and has been a major breakthrough in recognising what is needed to manage this disease. Research has conclusively shown that young people living with Duchenne who have access to multidisciplinary expert care are living ten to fifteen years longer and have an improved quality of life. In Denmark young men with Duchenne live well into their 30s and 40s – why is this not the case in the UK?”
Mr Catlin continued, “Last year Action Duchenne published its white paper ‘Time to Stop Wasting – An End to the Postcode Lottery’. This was based on findings from our nationwide consultation process where we spoke to many families affected by Duchenne. The process confirmed that many, in fact, over two thirds of parents feel that their children are receiving sub-standard medical care. Duchenne is a complicated condition, where expert medical care can make an enormous difference to length and quality of life. Currently there are only two Centres of Excellence in the country. We are calling for more Centres so that families can receive the medical care in line with the guidelines published, within a reasonable travelling distance of their homes. This is the very least we should expect, but we need Government support and increased funding to make this a reality rather than cuts in services, which is what they are currently threatening.
“Action Duchenne’s work through the Race Against Time Campaign was instrumental in bringing together scientists and researchers and two other charities, to raise awareness and funding for critical research. The results of the clinical trials conducted by AVI BioPharma in conjunction with MDEX* consortium in the last few weeks were extremely promising. We urgently need that crucial research to continue. For this we desperately need Government support and funding. The young people with Duchenne don’t have time on their side, we need this research to have secure funding to continue so that we can ensure that it can really make a difference.”
Notes to Editors
*About the MDEX Consortium
The MDEX consortium led by Professor Francesco Muntoni, is a multidisciplinary enterprise to promote translational research into muscular dystrophies, and is formed by the clinical groups of Professor Francesco Muntoni (UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle University), and scientists from Imperial College London (Professor Dominic Wells), UCL Institute of Child Health (Dr. Jennifer Morgan), Royal Holloway University of London (Professor George Dickson), Oxford University (Dr. Matthew Wood) and University of Western Australia (Professor Steve Wilton). In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participate in the Consortium.
For more information, visit www.mdex.org.uk.
About Action Duchenne
Action Duchenne (formally Parent Project UK) was set up by Duchenne families in 2001 to promote new research for a cure for Duchenne. The charity has a strong record in funding research and has to date funded 8 major projects costing £800,000 and has lead the £1.6m DoH funding of the MDEX project. These projects have enabled much needed early work to be completed on exon skipping and other therapeutic approaches.
Action Duchenne holds an international conference every year to bring together researchers and families to exchange new research developments and provide a vital meeting venue for scientists.
In 2005 Action Duchenne launched the Duchenne Registry, the first National Duchenne database that holds gene information of people living with Duchenne and can be used to speed up the recruitment of patients for clinical trials.
In 2006 Action Duchenne launched a comprehensive learning and behaviour toolkit for use by parents and education professionals.
For more information please visit: www.actionduchenne.org
Nick Catlin Andreina West
CEO, Action Duchenne PR Artistry Limited
Tel: 0208 556 9955 Tel: 01491 639500
Email: firstname.lastname@example.org email: Andreina@pra-ltd.co.uk
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