Each Year, We Set Huge Fundraising Goals
Many might wonder, “Why so much?” The Foundation’s mindset is to focus on research happening right now, as well as emerging scientific ideas.
We don’t wait for the next project that needs funding, they go out and find it (and sometimes even build it themselves). We collaborate and influence the pace of research for a cure.
And any possible treatment or cure requires pre-clinical research and work before it ever becomes a clinical trial for children. All of which requires funding, which is possible because of your support.
Our Fundraising History
How Are Your Donations Used?
Every single dollar matters, and adds up toward this effort and all net dollars go toward this urgent mission. That might be drug production for children to be treated, clinical trial resources, tackling the antibody suppression issue in gene therapy, or break-through research in enzyme replacement, stem cells, chaperone therapies.
Time is not on the children’s side, but HOPE is.
Your compassion is making the difference. You have our deepest thanks for your support for this life saving mission.
How You Can Help
There is a multitude of ways you can help. Find the one (are several) that suits you. Every bit truly helps.