Welcome to Cure MIOP
The Ryan Wersten MIOP Foundation was created in memory of Ryan Wersten who lived for only six months after being diagnosed with Malignant Infantile Osteopetrosis (MIOP). Children who are affected with this disease do not have proper working osteoclasts to break down bone in the body. As a result, these children’s bones continue to build up in their bodies without ever breaking down. This causes fractured bones, blindness, deafness, and ultimately it is fatal. At this time, the cure for MIOP is to attempt a bone marrow or stem cell transplant. This procedure is risky because it wipes out a person’s immune system, which makes the cure nearly as life-threatening as the disease.
First, we are supporting current research that is looking into a safer and more effective means of performing stem cell transplantation in children with MIOP. The Ryan Wersten MIOP Foundation has partnered with Dr. Paul Orchard, a physician-researcher at the University of Minnesota who is interested in exploring a strategy for correction of the patient's own cells through gene therapy. This has the potential to eliminate many of the risks associated with transplantation, and may prove equally effective. His initial studies will test methods of gene delivery to the blood stem cell, and the ability to restore the function of the defective gene associated with the majority of cases of osteopetrosis. Second, we support families who have a child with MIOP who is receiving a bone marrow transplant.
Ryan's story began on July 1, 2005. Ryan was born exactly 3 weeks early. It was a "normal" delivery with no complications. Early the next morning... Read more