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Science is Moving Fast...and so are We!
CureDuchenne brings a different perspective to DMD research. Many research projects are now seeing success in lab animal models. As our expertise has been in business, our goal is to identify research that has the most likelihood of making it to clinical trials and then provide the financial bridge that will take it from the lab and into human trials. We are thrilled that private industry has taken an interest in Duchenne, as they have the model and resources to bring it to market.
Some of our current and potential funding projects could help the entire population of DMD boys. Others projects are mutation specific and may initially serve a smaller segment of DMD boys. We believe that even if we have to save one boy at a time, it is worth it. If developers of insulin had waited until their therapies were as good as they are today, many people would have died unnecessarily. We know future generations of drugs will be better than the first generation. But without first generation drugs and therapies, the evolution to more effective treatments can't happen.
Three years ago when we founded CureDuchenne we immediately identified the exon skipping program that Prosensa and Leiden University were developing as a very promising research target.
After nearly a year of due diligence, we committed to fund Prosensa’s systemic delivery program for exon skipping in the amount of $1.3 million. At that time, we had a mere $10,000 in the fund. Today, thanks to the hard work and generosity of our supporters, we have completed this funding and are currently funding three other substantial projects.
Duchenne research is progressing quickly and from several different approaches. At the least, we want a drug that will offer the benefits of steroids without the negative side effects. Our hopes, dreams and prayers are for a true cure for our sons…while there is still time for them.
This last year, we funded over $1 million in Duchenne research. Our goal is to make larger grants to select investigators who have a solid business plan.
CureDuchenne wants to ensure that a research organization has adequate funds to thoroughly investigate and complete a project. We want to minimize or eliminate any delays to promising research that may be caused by funding issues. Paul and Debra Miller are the founders of CureDuchenne. From day one, they have worked tirelessly to make a difference. They do not take a salary. Their motivation is clear and compelling. Their 10 year old son has Duchenne. And more than anything, they want him to have…A Chance for a Lifetime.
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