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Band member with Duchenne muscular dystrophy hopes FDA acts fast on experimental life-saving drug

Posted at 6:00 PM, Sep 29, 2016
and last updated 2016-09-30 20:03:32-04

WESTMINSTER, Colo. -- Whether on the field or in the stands, teamwork benefits us all. 

Just ask Jack Knight of the Standley Lake Gators High School marching band.

“For me, I don't worry about the music as much. It's more about the friends you meet and the fun you have," he said.

Jack plays his baritone with one hand while navigating his wheelchair with the other, while his 9-year-old golden retriever Amber sits by his side.

“She goes everywhere with me,” Jack adds.

Jack is very active -- despite battling Duchenne muscular dystrophy -- a degenerative muscle disease with no known cure.

"It cuts their life expectancy,” is how Jack’s mom Angela describes the disease. “You're looking at teenaged boys and they're toward the end of their life.  They're past middle age at that point."

Angela Knight believes her son still has the use of his arms and hands at age 16 because he’s been taking the experimental drug Translarna as part of a clinical trial.

"What we know about the drug is that it has changed the course of Jack's life,” Angela said.  “He's able to play in the marching band at 16 and he's had absolutely no side effects over the course of ten years."

Translarna has been approved for use in Europe.  EU health officials believe the benefits outweigh the risks.  But in the U.S., the Federal Drug Administration (FDA) is not so sure and wants to see more tests.

Tests take time, something Jack and other boys with Duchenne don’t have.

"They would take generations and generations of boys dying with Duchenne to get the information that the FDA needs," Jack’s mom said.

Angela also fears that without some movement on the FDA's part the drug maker may give up -- denying Jack the medication that seems to be helping him.

She says she hopes leaders in Washington eventually use some common sense, to help extend the lives of young men like Jack.

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