New drug helping some children with Duchenne muscular dystrophy - ABC 12 – WJRT – Flint, MI

New drug helping some children with Duchenne muscular dystrophy

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(05/27/14) - It's hard to imagine the pain this mother must feel.

It's a dramatic story of a drug for one of her son's, and hope dangled before the other.

One uses the stairs, the other a ramp - both have the same disease - Duchenne muscular dystrophy, affecting mostly boys, and usually killing them before their 21st birthday.    

While 12-year-old Max is getting what some are calling a miracle drug, his 15-year-old brother, Austin, is not allowed to take the drug.  

"This is actually completely ludicrous," said Jenn Mcnary, Austin and Max's mother. "I know that my son will die if he doesn't get it."   

What Austin can't get is a drug called eteplirsen. He wasn't eligible for the drug trial because he was already in a wheelchair, which is where all Duchenne md patients end up. He has been in the chair since he was 10 and a half.

His brother was headed to the chair, too. Young Max was already using a walker, a cane, occasionally a wheelchair, but Max could walk six minutes on his own - that was the test. So he got into the drug trial three years ago. After weekly infusions of the drug, his disease progression was halted - and even reversed.

Max was walking better, running faster. getting stronger. His brother - weaker, watches from his wheelchair.    

"It is hard because it is there and I can use it but I'm not able to and Max is on it and he's getting better," Austin said.

"Some boys are running, some boys are now playing sports," said Christine Mcsherry, CEO Jett Foundation.

Mcsherry says never before in the history of this devastating disease has a drug performed so well - ending the inevitable.  

"Once those boys decline, they continue to decline, they go into a power chair, and then their respiratory status will start to decline as well," she said.

She knows - her 18-year-old son, Jett, is at that point.

Walking may be out, but boys in the trial are showing improved pulmonary function. She is certain getting Jett the drug now would save him.    

"There is either this for Jett, there is nothing else that is going to come down the pipeline fast enough to save his life, this is it," she said.

The FDA wants more study data, more information - more proof. Austin and Jett would need to take eteplirsen for six months at least to get it into their system, to begin making the dystrophin protein Duchenne patients lack.

At 15, Austin says he is not afraid to die, which could happen while waiting on the FDA to make a move.  

"Give my brother the drug now," Max said.

A desperate wait for these mothers, this brother, and this boy - and the medicine they need to live.

Members of the Duchenne alliance have been pushing hard for immediate approval of the drug from the FDA.

They collected more than 100,000 signatures so the White House would look into the issue now before another boy dies.
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