Montgomery County family pushes for drug approval
Updated On: Apr 04 2014 06:19:56 AM CDT
A Montgomery County mother is part of a nationwide fight to get a drug approved that she says could change the lives of thousands of children, including her own.
Lauren Fritz's two boys have a form of muscular dystrophy called Duchenne Muscular Dystrophy and there's no known cure.
Ten-year-old Jake and seven-year-old Ryan were diagnosed almost six years ago.
"It can be genetic but it can pop up in any generation and it just popped up in our generation so we don't have any family history," said Lauren.
"It's hard to get upstairs a lot of the time and I can't really get off the ground if I'm sitting down," said Jake, a fifth grader at Lower Pottsgrove Elementary School.
"There is no cure. The progression is 100 percent fatal," explained Lauren.
It is said to be the leading genetic killer of children, affecting one in 3,500 male births.
"The reality of this disease is by age 10 to 12 the muscle degenerates to the point where you need a wheelchair. In your teens you lose your ability to move your arms. You need help with all activities of daily living, basically you become a quadriplegic," Lauren said.
They've already had to make changes around their Upper Pottsgrove Township house including building a ramp leading into the house, getting a power scooter and wheelchair, along with a wheelchair-accessible van, and they are planning to make modifications to the bathroom.
Lauren said, "They take steroids which can prolong strength for a little while but the reality is there is no treatment to stop the progression of this disease and there is nothing that is approved right now that will keep them from dying."
But the Fritz family, and others out there, do have some hope.
"We know of a medicine that has been studied that could drastically change the outcome of this disease. It's called eteplirsen," said Lauren.
Supporters are pushing to have the FDA fast-track the drug's approval. More than 100,00 people have already signed a White House petition.
"The medicine so far has proven to halt the progression of the disease, it's proven to even regain a little bit of function in some kids and there have been absolutely no side effects," Lauren explained. "If that's approved in a short period of time they may not have to die from this disease."
"Life would be just easier," said Jake. "I hope we can get it."
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