We Believe a Cure Is Within Reach
Hunter Syndrome (MPS II) is a rare genetic disease that affects less than 500 boys in the United States. These boys are missing an enzyme used to break down cellular waste. When that waste builds up, it leads to progressive damage throughout their little bodies. Some never develop speech, but some boys learn like a typical child then begin losing skills and what they’ve learned at a young age. They eventually lose the ability to walk, talk and eat. Most do not live to see their teen years.
Gene therapy, a revolutionary new approach to treating genetic diseases, may provide the cure these boys desperately need. Researchers are in the final stages of developing the first gene therapy clinical trial for boys with Hunter Syndrome. The only obstacle is the substantial funding required for such a trial. Project Alive aims to fund the trials and bring us one step closer to a cure.