Thank you, everyone!
We have raised our goal of $10,000 for research by Jan. 31, 2015!
“The Duchenne Alliance is funding a pilot program to test gene therapy in patients with Duchenne muscular dystrophy. The gene therapy has been successful in another pilot study involving patients with Becker Muscular Dystrophy. As those studies continue, the Duchenne Alliance is pushing to add the gene therapy to include Duchenne muscular dystrophy, a more severe form of the neuromuscular disease.
“The funding — $500,000 in total — will be used by a team lead by Jerry Mendell, MD at Nationwide Children’s Hospital (Columbus, Ohio) to test follistatin gene therapy in a trial for Duchenne muscular dystrophy.” more>
How You Can Help
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy, like all muscular dystrophies, causes weakening of the muscles. DMD is a recessive genetic mutation that occurs on the X chromosome. As a result, it almost exclusively affects boys. Unfortunately for the boys who have been diagnosed with DMD, it is the most severe of all the muscular dystrophies:
- Boys with this disease are diagnosed earlier, usually between 3 and 5 years of age.
- They lose muscle function more quickly, many boys are in wheelchairs by 10-12.
- They die earlier, most boy’s with DMD die in their mid 20′s.
Since Gus’ diagnosis, many well-meaning people have tried to reassure us that they know someone in their 40’s, 50’s or 60’s with muscular dystrophy.
What We’re Funding
At Hope for Gus, we consider ourselves stewards of the money that people donate. We feel incredibly indebted to you, the donor for two reasons:
- We are so appreciative of the fact that you are willing to join us in the fight to save Gus and the tens of thousands of boys like him who are suffereing from DMD.
- We are honored that you have entrusted us with your charity dollar. We will do everything in our power to justify that trust. We take it very seriously because it comes down to saving lives.
We work to leverage every donation through the Duchenne Alliance. For example, if we are funding worthy research, we may challenge other foundations to match our donation, or we may work with others to match theirs. Our goal, of course is to bring promising research to fruition; to bring treatments to our boys.