Drug could help kids with Duchenne muscular dystrophy

BIRMINGHAM, Ala. (WIAT) — By 11 months, Scott Griffin knew there was something different about his son, Gabe.

“When he would crawl his arms would give out,” Griffin said.

By age three, he knew why. Gabe had Duchenne muscular distrophy, which is the top genetic killer of boys in the world.

“There is no cure. There is no treatment,” Griffin said. “There’s going to come a time that I’m going to have to look this little boy in the face, and I’m going to have to tell him that you’re dying.”

However, all hope isn’t lost. A new experimental drug, called eteplirsen, could change Gabe’s life and thousands of other boys suffering from the disease.

“The drug is safe. There’s not a single side effect” Griffin said.

The problem, though, is that the drug isn’t available yet.

Although the Food and Drug Administration (FDA) says it’s committed to making safe and effective drugs available to patients with the disease, the agency has refused to grant accelerated approval for the drug.

That move could make the drug available to patients by the end of the year.

That’s why Griffin and other parents are urging people to visit the website, theracetoyes.org, and sign a petition.

If they get 100,000 signatures by March 29, the White House could urge the FDA to grant accelerated approval of the drug.

2014 WIAT-TV CBS42

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