11 June 2009
Action Duchenne lobbies Government for ‘Best Practice’ standards of medical care for all and for funding to develop a cure for Duchenne Muscular Dystrophy
Parents and boys living with Duchenne march on Downing Street to raise awareness of heartbreaking disease and demand an 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 to No. 10 Downing Street to bring an end to the continuing serious under-funding for research into the condition. Results from recent clinical trials into the effectiveness of Exon Skipping as a gene therapy for the treatment of Duchenne have been very promising and scientists say that a cure is tantalisingly close, but more funding is needed. 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.
Duchenne Muscular Dystrophy currently has no cure. It is a severe muscle wasting disease that affects boys and young men, resulting in boys 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.
Prime Minister Gordon Brown stated on 31 October 2007, “The Government fully accepts that everything possible needs to be done to bring an end to the tragic impact Duchenne Muscular Dystrophy has on individuals and their families.” Since then there has been very little action from the Government. Following last year’s lobby, Action Duchenne will be calling on the Government to:
• Redress the historically poor levels of research funding over the last 10 years by allocating £30m of funding for new Duchenne research and clinical trials UK wide. To include further funding for the current UK MDEX* Exon Skipping trials.
• Ensure that all people living with Duchenne are given immediate access to new drugs and treatments
• Ensure that all people living with Duchenne are supported by a Centre of Excellence within 50 miles of their home (currently there are only two, one in Newcastle and one at Great Ormond Street Hospital in London), with access to multi-disciplinary teams trained in the treatment of Duchenne patients
• Ensure that all people living with Duchenne receive medical care in accordance with the internationally agreed Standards of Care for Duchenne Muscular Dystrophy
• Significantly improve the care received by patients as they reach adulthood.
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.
Carl Tilson who is living with Duchenne and is an active campaigner for Action Duchenne based in Manchester said, “I will be attending the Lobby of parliament on 16th June in London. I have been living with Duchenne for 22 years and now it has got to the stage where I am paralysed and can’t walk or even feed myself. Everything has to be done for me. Many of my Duchenne friends have died. The worst thing is living with this death sentence on my head. They have made great breakthroughs in medical research but lack of government funding for clinical trials is holding back a cure or treatment.”
Carl continued, “I want to say to Gordon Brown that I was pleased to hear him say that he would do everything to help us. But so far, we have seen nothing. Gordon, Is there anything more important to your government than saving young people’s lives?
We don’t want to be a burden on our parents and we don’t want handouts. We just want to live an independent life, going to college... We want to work and we want to contribute to society. Most of all we want our bodies to function properly to enjoy simple things like eating real food without the fear of choking or chest infections and being able to do things people can take for granted. We want to live without the fear of a bad cold turning to pneumonia and then to respiratory and heart failure. Give the research teams and clinicians the money to get on with the job of finding a cure and also do away with the unnecessary red tape which is keeping boys like me fighting for survival from seeing any improvements.”
Nick Catlin, CEO of Action Duchenne said, “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. However, we need to do much, much more. The results of the clinical trials conducted by AVI BioPharma in conjunction with MDEX* consortium at the end of last year was 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 start as soon as possible so that it can really make a difference.”
Mr Catlin continued, “Action Duchenne has recently published its white paper ‘Time to Stop Wasting – An End to the Postcode Lottery’. This is 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 a third 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 best medical care within 50 miles of their homes. This is the very least we should expect, but again we need Government support and funding to make this a reality.”
Action Duchenne has also arranged Lobbies to the Scottish, Welsh and Northern Ireland National Assemblies.
Notes to Editors
*About the MDEX Consortium
The MDEX consortium led by Professor Muntoni, is a multi-disciplinary enterprise to promote translational research into muscular dystrophies, and is formed by the clinical groups of Professor Francesco Muntoni (Imperial College London and 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 and Dr Ian Graham), Oxford University (Dr Matthew Wood) and University of Western Australia (Prof 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
CEO, Action Duchenne
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