Puyallup family hopes UW scientist can find cure for boys' Duchenne muscular dystrophy

Joshua France talks about his rare disease.

A local family knew they were facing an overwhelming challenge, when their son was diagnosed with rare, muscular disorder.

It quickly takes away your ability to walk and eventually takes your life. Then, doctors told them their other son would suffer the same terrible fate.

All they want is a cure. A scientist from the University of Washington is working to find one.

For the France family from Puyallup, going to the park takes a bit longer, because 11-year-old Joshua has Duchenne muscular dystrophy.

“When I'm in a wheelchair, you can't do much -- that teaches you to have more patience, that teaches you to be much more humble,” Joshua said.  

His 8-year-old brother Samuel also has the disease.

“I struggle with having to watch them go through it, to see your own child suffer, to know there's nothing you can do about it is the hardest thing,” said their mother, Christine France.

The disease is caused by a genetic mutation. It's only found in boys. – one in 3,500 -- causing their muscles to become weak.

Dr. Jeffrey Chamberlain of the University of Washignton said, “It can be a very devastating disease. The kids usually lose their ability to walk in their early teen years. And they show progressive loss and wasting of muscle function throughout the rest of their life.” 

Chamberlain is one of the leading Duchenne researchers in the world.

"When I first started working on this disease a little more than 20 years ago, the life expectancy was in the late teens, now as I mentioned, many of the patients are living well into their 20s and well into their 30s.”

In his lab at UW, Chamberlain and his team are making synthetic genes that can be injected into the muscles of a person with disease.

“The cause of the disease is that a key protein is not being made, if we can figure out a way to replace that protein, we can have a major impact on the disease." 

While he says a cure is unlikely in the near future, they hope to block the disease from progressing. And if treated in young children, they may be able to prevent it from taking hold.

The possibility, however, gives the France family the reason to hope for a cure.