This is an important debate through which I hope to raise awareness of the serious medical condition, Duschenne Muscular Dystrophy.

Duchenne Muscular Dystrophy is named after Dr Duchenne de Boulogne who worked in Paris in the mid-19th century and was one of the first people to study the muscular dystrophies. The problem is known to result from a gene defect in a single important protein in muscle fibres called dystrophin. Without dystrophin to strengthen the muscle cells they become weaker and eventually waste away.

Duschenne Muscular Dystrophy (or DMD) is the most common, the most progressive and the most deadly type of Muscular Dystrophy. It is an X-chromosome linked disease, and as such predominantly affects boys. It affects one in 3500 boys worldwide.

Duchenne Muscular Dystrophy is usually diagnosed when a child reaches 2-3 years of age and does not reach developmental milestones such as standing and walking. As the boys progress through childhood they are often prone to falls and eventually are unable to walk, usually needing a wheelchair by the age of 11.

Through the teen years a boy will become progressively disabled as the muscles seriously weaken and waste away. In the final years of the boy's life 24-hour care will be required including night-time ventilation and feeding support. A young adult with Duchenne Muscular Dystrophy is unlikely to live beyond their 21st birthday. Death is usually caused by heart or lung failure as the muscles supporting the vital organs finally become so weak they are unable to maintain life.

DMD is what is known as an ‘equal opportunity' condition. Although many cases of Duschenne are hereditary, an increasing number of cases are caused by spontaneous mutations. As such, the condition could cast its shadow over anybody of any background or nationality. In more than one sense, it is a case of ‘genetic roulette.'

I was first alerted to DMD earlier this year when two constituents of mine from Cardiff West - Nick Catlin and Janet Hoskin - approached me for help. Nick and Janet have a two-year-old son called Saul. Saul was diagnosed at the unusually young age of 5 months with having DMD after a routine blood test for liver function.

Saul is the Catlin's first child – although, if you will allow me the luxury, I would like to take this opportunity to congratulate them on the birth of their second child, a girl named Amelia, last week, and I wish all of them best wishes for the future.

As I was saying though, Saul was the Catlin's first child and, I think it is fair to say, that the experience and the acceptance of Saul's diagnosis was devastating.

Writing in February, Nick told me that they thought that ‘There is a death sentence on the head of my son.. A cure is not an impossible dream. We need more resources and facilities.'

In the same letter, Janet added that they wanted the Government ‘to fund research into DMD so that our sons are given the chance to live full and happy lives.' At present, Saul is a healthy, buy toddler, more concerned with cars and diggers than health matters. It is very hard to believe that such a vibrant little boy will be condemned to such a short life, never being able to fulfill his potential.

The plight of their son – and the intense desire to find a cure for the condition – led Nick and Janet to form their own charity – Parent Project UK (or PPUK for short) - in February of this year. One of the main spurs behind the establishment of PPUK was the passing of the Muscular Dystrophy Community Assistance, Research and Education Act through the United States Congress on 21 December 2001, effectively ringfencing millions of dollars of research into DMD.

The main aims of Parent Project UK are to raise awareness of Duschenne, to raise money for research and, like any good lobbying group, to put pressure on the Government to release funds in an attempt to find a cure. At present, only a limited amount of Government money is earmarked for research into this specific area, and scientists are forced to compete for funds with research into other serious illnesses.

Since the charity was founded, Nick, Janet and the other helpers of PPUK have worked long and hard to raise both awareness of (DMD in Wales) and also extra funding to pay for new research. Nick and Janet were lucky enough to receive a start up grant of £5000 from the then National Lottery, and on Saturday 27 July this year, members of the Royal Monmouthshire Engineers Field Support Squadron took part in a relay, organised by PPUK, between Swansea and Cardiff. To date, this run has raised £2000. PPUK has also successfully bid for a grant of £48,950 from the Community Fund, which, over the coming year, will cover the costs for a project co-coordinator and general running costs of the charity.

As a relative newcomer on the scene, PPUK is following in the footsteps of two other charitable and lobbying organisations: The Muscular Dystrophy Campaign and the Duschenne Family Support Group.

The Muscular Dystrophy Campaign is a national charity focusing on all muscular dystrophies and allied disorders. It has pioneered the search for treatments and cures for over 40 years and also aims to provide medical and emotional support for sufferers and their families.

The Duschenne Family Support Group was founded in 1987 and it is a national charity that was founded and is run by families affected by DMD for families affected by DMD. The Group strives to bring families together for mutual support, sharing of information and social activities.

In recent months, the three charities have pooled their resources to co-ordinate a nationwide campaign entitled Right to Survive. The campaign aims to increase funding for research into finding a cure for Duchenne Muscular Dystrophy in order to give sufferers the right to survive into adulthood.

The charities are requesting that government money be dedicated for research into exploring treatments for this disease. Potentially children born now with Duchenne muscular dystrophy could have the chance to a long life.

The UN Convention on the Rights of the Child, adopted by the UK in 1991 states that the government commits to the right to life, survival and development of children. The Right to Survive campaign calls in this pledge in order to give boys with Duchenne muscular dystrophy the right to survive into adulthood.

The campaign has operated a three-pronged attack in its attempts to raise awareness:

I tabled Early Day Motion 1341 on 20 May this year, and it has so far attracted 85 signatures from Members on all sides of the House. I hope that as a result of this debate today, that that figure will rise in the days following.

The Right to Survive Campaign has also been gaining pledges of support from members of the public, and I will be taking members of the campaign to Number Ten this afternoon to hand in these petitions of support.

The third prong of the campaign is taking place tonight, when Ian Gibson and I will be hosting a briefing in Committee Room 11, where Members of both Houses will be given the opportunity to discuss the issues surrounding DMD with campaigners from all three charities as well as sufferers and family members. I urge all Members of this place and the other to spare time this evening to come to the briefing and find out more about Duschenne Muscular Dystrophy first-hand.

Now of course, the other main aim of the campaign is to try and persuade the Government to release extra funding for research into a cure for DMD.

At present, the Muscular Dystrophy Campaign is the only voluntary organisation in the UK funding scientific research into finding potential cures or treatments for all types of muscular dystrophy and related disorders. The charity supports a programme of around 30 research projects around the UK and in Continental Europe at an investment of £1,500,000 in 2002. These projects are working to characterise the genes and proteins responsible for the individual types of muscular dystrophy as well as investigating techniques for potential treatments and cures. Results from these projects are shared around the international science community to ensure knowledge is shared effectively on the range of initiatives under investigation.

The main agency through which the government supports medical and clinical research is the Medical Research Council (MRC). The MRC is an independent body which receives its grant-in-aid from the Office of Science and Technology, and is, as such, under the auspices of the DTI.

MRC spending on muscular dystrophy research for 2001-2 is an estimated £950,000 which represents 0.24 per cent of it's total £394 million gross expenditure. This proportion of funding has been consistent over the past six years. In addition, the Department of Health provides support to the NHS R and D budget to assist research conducted within the NHS.

At current levels the voluntary sector is funding a third more research than the government. Quite rightly, I think, the charities find this position unacceptable and are calling for increased funding into research which is ringfenced for Duchenne Muscular Dystrophy. I would like to take this opportunity to ask my Hon. Friend, the Minister, what, if any plans, the Government has in this area?

Any change in Government funding will also require a shift in strategy from the MRC as the position is currently one of limited funding for projects associated with rare conditions. I believe that researchers currently working in the field of muscular dystrophy often propose grant applications, many of which receive the highest possible ranking from the MRC, but are then turned down on precisely those grounds. As part of the ongoing campaign, PPUK, the Muscular Dystrophy Campaign and the Duschenne Family Support Group intend to meet with the MRC to discuss ways of overcoming this funding issue and to prepare the ground for potential clinical trials in the UK.

I hope that this issue can be resolved smoothly and speedily, as I believe that too much time has been lost already. At the end of the day, there are lives in danger here and I would hate to think that they are being further imperiled for the sake of administrative niceties.

One other issue I would like to raise regarding DMD is the method of genetic research employed. The exact role of dystrophin within the cell is not fully understood and is something that scientists are trying to determine. One of its roles is thought to be structural due to its position within the muscle cell membrane where it forms part of a complex known as the dystrophin-glycoprotein complex (DGC). Dystrophin forms a link between the outside and the inside of the muscle membrane. Without dystrophin the membrane becomes weak, breaks down and the muscle cell dies.

The main area of research aims to pinpoint exactly why, in certain cases, the dystrophin is absent. There are three main avenues of research: muscle cell transfer, gene therapy and more general pharmacological research.

Muscle cell transfer utilizes genetically-modified self cells. Research undertaken by Dr Di Watt using experimental models, has shown that under conditions where skin cells are grown up outside of the body in a liquid medium that previously contained muscle cells, some of these skin cells convert to muscle cells. Research is ongoing in the factor responsible for this conversion – a reaction referred to as Galectin-1 - and to gain a better understanding of the type of cell that is capable of this conversion - not all skin cells have this ability. Should this have therapeutic potential, skin cells from a DMD person could be removed, a correct version of the dystrophin gene inserted, these cells grown up and then re-introduced into the person where the cells would help to repopulate the muscle and form functional muscle tissue.

The main advantage of this form of research is that skin cells can be used instead of muscle cells, which are substantially reduced in number in cases of DMD.

Gene therapy or, to use its more technically accurate description of gene repair technology, looks at ways of repairing the gene either in situ, or outside the body and then putting these cells back. This new technology can use repair mechanisms normally present in cells to target specific mutations and correct them. This system does not involve introducing a new gene into cells it repairs the existing faulty gene. It can only be used on very small mutations (one letter of the gene code wrong), which accounts for approximately 15% - 30% of individuals with DMD.

Both of these types of research are dependent upon stem-cell research and I think that it is important that the Government's decision to continue with genetic research is to be applauded. It is a notable, if gloomy irony, that the United States has ring-fenced money for DMD research but has, at the same time, called a moratorium on stem-cell research.

It would be a tragedy if progress towards a cure were to be held up because of a lack of resolve in the US Administration - when funds were available – that is why it is important that funding is available in the UK for stem-cell research.

Pharmacological approaches to DMD treatment encompass a more traditional range of studies including the utilization of steroids and metabolic stimulants which aim at improving mobility as opposed to eradicating the problem in toto. Any satisfactory treatment is likely to draw upon all three forms of current research, as it is hoped that these three approaches will compliment one another and eventually provide a cure.

Every member of this House is likely to have a child like Saul living in his or her constituency. Every one of us bears a responsibility to do all in our power to find a cure for this dreadful disease. I hope that the Government will confirm that it supports research for these so-called rare diseases – and in particular will make sure that public policy supports the kind of medical research which is most likely to produce a cure for Duschenne Muscular Dystrophy.