STRUGGLE AGAINST MUSCULAR DYSTROPHY (SAM)
Thank you for taking the time to learn more about Sam, his condition and the charity that we set up to raise funds to help find a cure for muscular dystrophy.
Sam is a gorgeous little boy with blonde hair, big blue eyes, and a mischievous sense of humour. Everyone who meets him falls in love with him. In so many ways he is like any other little boy, except for the fact that his muscles do not work properly. This is because Sam has a progressive muscle wasting disease called Congenital Muscular Dystrophy.
Sam was diagnosed with this condition at just 13 months when he showed signs of delayed milestones. The doctors tested his blood, and then we had an agonizing wait for him to have a muscle biopsy, which confirmed everyone’s worst fears. We were told that Sam’s muscle sample showed a problem with the production of a protein, needed to keep muscles healthy. I could not even begin to take in the information at that stage.
Muscular Dystrophy is a group of over 40 neuro-muscular diseases affecting thousands of children and adults in the UK, Ireland and across the world. The disease comes in many forms and causes the muscles of the body to gradually degenerate and waste. Children often become dependent on wheelchairs at a very early age and as the muscles continue to deteriorate they might become quadriplegic, suffer from scoliosis (twisting and distortion of the spine) and develop painfulmuscle contractures. Many eventually lose the ability to breathe and eat without assistance.
When Sam was diagnosed with Congenital Muscular Dystrophy I asked the doctors to tell me something – anything - to give me hope… but right now there is no cure. There is not even a treatment to slow down the progression of this horrible disease. There is no government funding – no big research lab – no multi-national pharmaceutical company just waiting for the nod. As a rare disease, the form of muscular dystrophy from which Sam suffers has, until now, been largely ignored in research and drug development. I knew then what a battle we would have to fight even to raise people’s awareness of CMD, let alone move us along the path towards finding a cure.
All of us were shocked to the core when we learned of Sam’s prognosis. In April 2008 a small group of friends and family set up the charity “Struggle Against Muscular Dystrophy” to help raise awareness of the condition and generate funding for research. To date, with huge support from the people of NI and a number of extremely successful fundraising events under our belts, we have raised almost £400,000!
One of our core commitments from the get-go was that we would establish and run as a ‘zero cost’ charity – in other words everyone involved with the charity would give freely of their time, expertise, products and services so that we could be in a position to promise faithfully that every penny we raise can go to research to help bring an effective treatment and eventually a cure one step closer not just for Sam, but for the thousands of other children whose lives have been devastated by this disease.
Every penny raised so far has been directed to international research projects through a tendering process, which we set up with clinical advisors who have painstakingly evaluated the merits and prospects of each grant application. This research has already moved us - slowly but surely - towards a potential drug candidate as a treatment for CMD. There is great cause for optimism that within the next 5 years we will at least have an effective treatment to slow the rate at which the muscles are wasting. This will potentially buy us a little more time for the scientists to find a cure – and that dream ignites and sustains the passion I have for raising the vital funds that will help maintain the research. I dream that Sam and all the other children who suffer from CMD will have a real chance of having a brighter future than the one currently predicted for them.
This will only happen with your help…
Thank you – on behalf of SAM and the many thousands of families in the clutches of this cruel disease.
You give us hope.