DMD has changed our lives.
DMD is short for Duchenne muscular dystrophy.
Duchenne is a genetic mutation that affects 1 in 3500 boys and in very rare cases it can affect girls. This mutation is specific to the X chromosome and is often passed down through the mother who is a carrier.
DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptoms usually begin between ages 3 and 5. Without the dystrophin protein in your muscle cells, they are fragile and easily damaged.
Historically, boys with DMD usually do not live much beyond their teen years. The ability to walk is lost in the early teens and life expectancy is shortened significantly.
At this point, DMD is always fatal and causes major stress and difficulty for affected families. There is no cure or treatment available to reverse the effects of DMD. The current standard of care for boys with DMD includes regular corticosteroid treatments and basic vitamins and mineral supplementation.
There are many promising clinical trials in the works and we pray for a cure to be found soon.
So, What can we do in the mean time?
While we wait for a cure, we will work to make life enjoyable and fulfilling for DMD boys and their families. You can help us do that.