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Breakthrough Drug For Duchenne Muscular Dystrophy

SPRINGFIELD, Mo. -- Duchenne Muscular Dystrophy affects 1-3,500 kids around the world and has a 100% fatality rate. However, this new drug could be the key to stop progression of the disease.
SPRINGFIELD, Mo. -- Duchenne Muscular Dystrophy affects 1-3,500 kids around the world and has a 100% fatality rate. However, this new drug could be the key to stop progression of the disease.

Arthur and Becky Underwood say their life changed 5 years ago when their son, Cole, was diagnosed with Muscular Dystrophy.

"It was tough when we found out, we thought something was wrong, but when we found out, it really made it tougher on us."

Cole is a 4th grader at Ash Grove Elementary. While he can still walk on his own, his parents say he has troubled doing what every 10-year-old should do.

"He likes to act normal, he thinks he can do everything like every body else, he likes feeding cows."

But as Cole gets older, he will end up in an electric wheelchair, unless doctors find something to stop the muscle deterioration.

"He knows he gets tired easy and that he can't walk as far but he doesn't really know about the disease that much."

That’s where the Race to Yes could help save his life. The campaign pushes for the FDA to approve a drug that builds protein and could help stop the progression. Dr. Jim Collins, a pediatric neurologist from Mercy Kids, explains.

"The theory was that it would continue to make a protein that may not be as functional as normal protein but still functional enough to do what the kid needs to do or at least stabilize the progression of the disease," says Dr. Collins.

Its still in the trial stages but has proved to help with the disease.

"The data on those patients looks good but again its only a small percentage of the population."

Dr. Collins says the drug would only be used for patients meeting a certain criteria.

"Its specific to the gene mutation that the child has and there’s multiple different types of gene mutations in the dystrophian gene."

While its not a cure, it could save the lives of children suffering from the disease around the world.

"If treated earlier, it may keep the child at a much higher functional level."

As for Cole's parents, they are hopeful the drug will be approved before its too late.

"The longer he walks, the longer he can do things for himself."

If you would like to sign the petition or learn more about the drug, go to TheRacetoYes.org. The deadline to sign that is March 29th. Right now, it needs more than 66,000 signatures.
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