Push To Fast Track Drug To Help Children With Deadly Disorder

By
UPDATED: Friday March 21, 2014 12:48 AM

It's a deadly disease without a cure, but researchers say they have a drug that slows the progression.

Now, families of boys with a rare form of muscular dystrophy are pushing for government approval.

7-year-old Jackson Maynard can play football with his big brother right now, but he likely won't be able to in a few years.

"He's starting to fall a lot at school, and he has difficulty getting up and down the steps," Jackson's mother, Kelly Maynard, said.

Maynard said her son was diagnosed with Duchenne Muscular Dystrophy two years ago. She said her little boy is losing strength every day.

"Boys are usually in a wheelchair by the time they are 10 or 12. It is a 100% fatal disease, most boys don't live past their 20s with this disease," Maynard said.
     
Researchers, including a doctor from Nationwide Children's Hospital in Columbus, recently went to Washington to testify on behalf of the drug Eteplirsen. Dr. Jerry Mendell said the drug works.

Maynard isn't able to give the drug to her son because it still hasn't been approved by the Food and Drug Administration. In an effort to change that, she is pushing a petition to get the FDA to accelerate approval for Eteplirsen.

Maynard said the goal is to get 100,000 signatures by March 29. She said only then will the White House comment as to why the FDA isn't moving faster.

In a letter from the agency's drug information director, Catherine Chew, she explains the FDA has reached no conclusions about the possibility of using accelerated approval for any new drug for the treatment of Duchenne Muscular Dystrophy. Maynard says she fears approval may be years away.

"In two years, a boy who needs this drug could lose the ability to walk or lose the ability to feed himself," Maynard said.

This particular drug will help about 13% of boys with Duchenne Muscular Dystrophy, while Jackson Maynard isn't one of them, his parents said they are fighting because this could open the door for treatment of other strains.

Read more about Duchenne Muscular Dystrophy:
http://mda.org/disease/duchenne-muscular-dystrophy

Petition:
http://theracetoyes.org/

©2014 by 10TV.com. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.