Duchenne muscular dystrophy (DMD) is one of the rarest diseases on Earth. Children are born with it - only once in every 3,500 live births (mostly in boys). In the United States, with a population of over 330,000,000 people, only about 20,000 children are stricken with Duchenne. 

And there is no cure.

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This is not the usual type of muscular dystrophy about which we do hear on TV telethons, etc. Most patients with muscular dystrophy manage to live until at least middle-age. And some even make it to old age.

But Duchenne muscular dystrophy is different. Without a cure, every one of these 20,000 children will die without even making it to adulthood. And their families will suffer the agony of watching them die very slowly. Because Duchenne is a progressive, muscle-wasting disease. And it shows no mercy.

By 11 or 12, these kids start losing the ability to walk. By the early teens, they’re in wheelchairs. By the mid-teens, they’re breathing on ventilators. And by the late-teens to early-twenties, they’re in graves. After suffocating.