Imagine your child is dying and no one can help. This is the case for tens of thousands of kids around the world who have one of ~3,000 known genetic ultra-rare diseases. The prevalence of each disease is less than 1 in 1,000,000 and when the disease affects 2,000 or fewer kids, pharmaceutical companies or public healthcare have no interest developing a treatment. 

With determination, and help from sponsors like you, we fund medical research to develop treatments for children with ultra-rare, fatal diseases. Treatment for one disease, Aspartylglucosaminuria (AGU), is ready for children. We need extra funds to pay for the drug to treat the kids. 

Your generous support will help save a child's life.