Please help Michael's Cause along with Ryan's Quest, Powers Promise and Pietro's Fight:
Duchenne Muscular Dystrophy (DMD) is the most common fatal genetic disorder diagnosed in childhood, affecting nearly 15,000 children and young adults in the United States with 20,000 new cases diagnosed worldwide each year. The patients are missing a gene, Dystrophin, which is essential for healthy muscle function. Without it, muscles deteriorate that support skeletal muscle strength, heart function, and breathing. Muscle weakness begins to develop in early childhood, and most patients are confined to a wheelchair by their preteen years. Loss of bone (osteoporosis) also occurs, which leads to fractures and further disability. In their late teens, they begin to develop breathing problems and heart failure. Many patients end up on breathing machines. There is no safe and effective treatment to alter the course of this disease, and most patients die in their late 20s to early 30s. The disease mainly affects boys, and there is no ethnic group that is spared with the same disease prevalence throughout the world.
Our sons, Michael, Pietro, Ryan and Trent, all suffer from this rare and fatal disease. We have each set up a non-profit foundation to find new therapies, and have come together as different entities to support the potential for the potentially life saving drug, OPG. This unique drug has brought us together because it has demonstrated impressive therapeutic effects. In addition, unlike many other drug candidates, it has the ability to help all of our sons, regardless of the genetic abnormality that leads to the loss of the Dystrophin gene. We have seen many potential drugs, and OPG is one of the most significant we have seen. We believe this potential therapy could be a big step forward in the treatment of DMD.
Michael, Pietro, Ryan and Trent are just a few of the many boys who are fighting for their lives each day. Together we won’t stop until every one of them is afforded a chance at a better and longer life. The boys’ muscle cells are dying every day, and so we are racing against the clock. Time is of the essence!
With your help, our foundations, Michael's Cause, Pietro's Fight, Powers Promise and Ryan's;s Quest, can honor our commitment to help fund this particular research project that has the potential to be life altering for our children.
About our Project: The money raised from this fundraiser will help support the completion of OPG-Fc animal studies in mice that have this disease (mdx mice), and allow us to begin to develop the production process of the drug candidate that will be used in its manufacturing and a Phase 2 clinical trial. Osteoprotegerin (OPG) is a hormone that first showed benefits in bone health when used to treat patients with osteoporosis. OPG has also been recently discovered to improve muscle strength in animals that are missing Dystrophin, and have DMD (mdx mice). The preclinical results are impressive, resulting in complete restoration of strength in the most affected muscles in DMD. OPG is thought to act not only by reducing inflammation and damage to muscles, but also by directly increasing the power of each muscle contraction to improve strength. OPG has dramatic effects on bone and muscle, including the diaphragm where damage is responsible for one of the two leading causes of death in DMD. The drug brings together many of the qualities of an ideal treatment for Duchenne, and has the potential to make a real difference in patients’ lives.
Please consider making a donation to help push this drug through clinical phases and help save our children.
Dave Schultz- Ryan's Quest
Kimberly Powers- Powers Promise
Manni Scarso- Pietro's Fight
Rob Capolongo- Michael's Cause
All funds go toward the mission of our non-profits 501c3. Donations are tax deductible.