Our mission is to save our son and other boys like him. There are 318 million people in America but only 20,000 males living with Duchenne Muscular Dystrophy. My son is only 1 of 119 other boys living with his mutation.
Duchenne Muscular Dystrophy is a genetic killing disease caused by their body’s inability to produce Dystrophin.
Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane. Without this ability to produce Dystrophin, they boys cannot produce skeletal muscle tissue. They end up in wheelchairs by puberty and on ventilators in their late teens.
- Short term goal: change the statistics of the age the boys lose their ability to walk.
- Our long term goal: is to creat a gene therepy that will save the life of anyone born with Duchenne Muscular Dystrophy.