Dr. Keith Foster, commented, “If this approach to treating muscular dystrophy is successful it will treat all patients with the condition. Currently, the most promising treatments are only suitable for a sub-set of patients. This could be a major breakthrough in the treatment of muscular dystrophy.
“Furthermore, if successful it is envisaged that the treatment of patients would ideally take place very soon after diagnosis, before there were any symptoms, which could enable the children to benefit maximally. Top up treatments may be required only every 5 years or so. This approach is significantly different to the current exon skipping therapies being developed where patients will need continual treatment.”
Nick Catlin, CEO of Action Duchenne said, “We are very excited about this latest development in the race to find a cure for Duchenne Muscular Dystrophy. This is the first treatment that could be called a cure, and while it is necessarily a very complex process, which if successful, will take a number of years to bring to market, it is too good an opportunity to miss”
“Action Duchenne is committed to finding a cure for Duchenne, however, we also recognise that while a cure is being developed we need to work with whatever resources are available now to make life as rewarding as possible for our children with this devastating, life limiting condition. This includes supporting research to find treatments that allay the symptoms of Duchenne, campaigning and lobbying for the best, multi-disciplinary care for all Duchenne patients which has been proven to extend life expectancy and Quality of Life, to develop educational and support programmes for Duchenne families to help them get the most out of life and to provide advice, guidance and support for all those living with Duchenne.”
Action Duchenne has supported research programmes to develop Exon Skipping gene therapy – which enables a damaged dystrophin gene to produce reduced amounts of dystrophin, Utrophin upregulation - a potential replacement for dystrophin and various drugs that treat symptoms of Duchenne, including muscle growth hormones, a drug to support the heart and a potential anti fibrotic drug.
Duchenne Muscular Dystrophy affects 1 in 3,500 male births in the UK, and is the most common and severe type of muscular dystrophy. Until fairly recently life expectancy for those living with Duchenne was late teens/early twenties. With the development of multi-disciplinary care this is improved to a point where young men are now living often into their thirties.
Notes to Editors
About Action Duchenne
Established in 2001 Action Duchenne aims to support and promote innovative research into a cure and effective medicines for Duchenne/Becker Muscular Dystrophy. The charity, which is led by Duchenne families, aims to promote awareness of the condition, to improve care services, and provide access to a range of educational and support/development programmes for people living with Duchenne at every stage of the condition. This is achieved by working in partnership with government agencies, NHS and care organisations, other charities, academic, scientific and research groups, and biotech companies worldwide.
Action Duchenne has consulted 26 members of our National Advocacy Council that includes young people living with Duchenne, parents and family members. The Charity has audited over 20 UK muscle centres and received responses from leading neuromuscular consultants on the extent of current care provision for Duchenne (www.dmdcentres.org)
Action Duchenne is actively campaigning for best practice in care for Duchenne based on the publication of an international consensus document in Lancet Neurology in Jan/Feb 2010. The published article was the product of an extensive review by 84 international experts in Duchenne Muscular Dystrophy diagnosis and care, and was supported by the Centers for Disease Control (CDC) in the US. It provides an expert guide to recommendations on the multidisciplinary care that should be available to all individuals with Duchenne. Several Duchenne patient organisations including Action Duchenne and the TREAT-NMD network have helped to produce a ‘Guide for Families’, based on the original academic article, which is written in language accessible to all without a medical background.
Since 2003 Action Duchenne has provided £4m for research projects and partnerships. The charity has worked with the MDEX consortium, Department of Health, and the Medical Research Council to deliver new clinical trials for Duchenne drugs. In addition, it has been instrumental in developing projects with biotech companies both in the UK and US including key projects with AVI Biopharma and Summit.
In 2005 Action Duchenne launched the DMD Patient Registry, a national database that holds gene information of people living with Duchenne that is used to speed up the recruitment of patients for clinical trials. For more information please visit: www.dmdregistry.org
In 2008 Action Duchenne launched the Include Duchenne project in partnership with Decipha. This programme addresses the learning and behaviour needs of those living with Duchenne. In 2011 Include Duchenne won the National Lottery Award for Best Education Project for Decipha. For more information please visit: www.decipha.org
Action Duchenne promotes social inclusion within the community for young men living with Duchenne through its Genius programme. For more information please visit: www.actionduchenne.org/
In 2010 the National Advocacy Council was launched to spearhead the lobby for government funding for research into Duchenne and to improve access to Standards of Care. www.actionduchenne.org/
Patrons of Action Duchenne include: actress Helena Bonham-Carter, cellist Julian Lloyd-Webber, journalist & broadcaster Martin Bashir and radio broadcaster & media trainer Susan Bookbinder. In addition, Richard Goodman, one of Great Britain’s leading young athletes is an Ambassador for Action Duchenne.
For more information please visit: www.actionduchenne.org
Tel: 0208 556 9955
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