WHAT HAVE WE ACHIEVED SO FAR?


 Adelina, age 2 We appreciate how challenging times are, and we know that it can sometimes be difficult to find those extra pounds to donate to charity, especially when there are so many worthy causes. That's why our 'zero cost' ethos is so critically important. Everyone involved in the charity gives their time, expertise, products and services for free. No money is taken from your donation for overheads, salaries or administration costs. This allows us to utilize every pound for the cause - to move us closer to treatments and eventually a cure for Congenital Muscular Dystrophy.
 
Thanks to the generosity of the people of Northern Ireland, SAM has already raised almost £200,000. This money is already being put to work.
 
Thus far SAM has funded the entire set-up and year one running costs for the CMD International Patient Registry. This registry, which was launched to CMD patients, consultants and clinicians in August 2009, aims to capture vital information on disease subtypes and genetic mutations, and will eventually provide researchers and scientists with information on disease progression and subjects for clinical trials. This is a vital first step towards our goal of finding effective treatments for the CMDs.
 
In January 2010 the first SAM Translational Research Grants were awarded. Four promising research projects are receiving funding from SAM totalling over £175,000 to evaluate 3 promising CMD drug candidates: an NFKappaB inhibitor, laminin 111, and a muscle specific IgF upregulator. A separate effort to identify disease biomarkers specific to merosin deficient CMD (MDC1A) will also be funded by SAM. Each application received during this year's grant process was subjected to a rigorous review process that included outside peer review and Scientific and Medical Advisory Board evaluation. 

"We believe that a focused investment in science will lead to CMD treatments which will bring hope to the many thousands of families whose lives have been affected by Congenital Muscular Dystrophy" announced Gillian Garrett, Chairperson of SAM. It is critical that we provide this level of financial support not just in year one, but also on an ongoing basis over the next 5-7 years in order to ensure that we reach our goal and maintain impetus.

 
WHEN WILL WE HAVE A CURE?


 Liam If only we knew the answer to that question! Research is currently concentrating in two main areas. Firstly, in the short term (2-5 years), we aim to support research into finding treatments for CMD - i.e. something to slow down the progression of the disease. A cure is the eventual goal, though this may well take another 5-10 years to come to fruition.
 
"The work we are doing in raising money to fund research through the SAM charity gives me hope. 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." (Tracy McCausland, founder of SAM)