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Concerned Mother Says FDA Might Remove Drug Helping Teen Sons Fight Rare Disease

PHILADELPHIA (CBS) — An experimental drug has been a life saver for two brothers in Chester County but the FDA might take it away.

Two teenage brothers have a rare genetic disease. They've been doing well with this experimental drug for years, but the FDA says there's not enough evidence it works.

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Like most brothers, Elliott and Henry Johnson are competitive and love video games but the Downingtown brothers have trouble walking, so they usually rely on scooters. They have Duchenne muscular dystrophy, an incurable progressive disease.

"You can't do other stuff your friends can do and stuff. And you're different and stuff like that," Elliott said.

Elliott was diagnosed first, ten years ago, and Henry received his diagnosis a month later.

"It was the worst day of our life as parents," their mother, Joanna, said. "The dreams you have for your children just go up in smoke. You're lost. We were struggling to cope we didn't know what to do."

Joanna and her husband Paul were told their children with Duchenne would slowly lose their lives, but there was a ray of hope.

"The doctor called us and said, 'I have the worst possible news but I have the best news in the worst possible case scenario and that there's a drug trial,'" Joanna recalled.

The drug Ataluren claims to slow the progression of Duchenne. It quickly gave the Johnson brothers more mobility.

"It's a life changer for them," Joanne said. "It was like you were seeing a miracle in front of your eyes."

After school, every day, they're in the backyard playing with their dog, but their fun days are now in jeopardy.

The FDA has rejected approval of the drug that keeps them moving. If the appeal fails, the drug will no longer be available.

"I'm terrified," Joanne said. "I'm absolutely terrified that all the benefits that we're seeing are just going to go away, then we're back to square one with no hope and no help, nowhere to go."

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In rejecting the approval, the FDA said there wasn't enough evidence to prove the drug works.

"I understand they (the FDA) want more data," Joanne said. "I don't have time. The boys don't have time to wait. You have 12 years with no safety concerns whatsoever."

Because it's a rare disease, the scope of the drug trial was limited and while it might not have worked for others, it did for these two brothers.

The FDA is expected to have a decision on the appeal around Christmas. The Johnsons are hoping they have something special to celebrate.

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