KINGSTON (CBS) — A Kingston mother is in Washington D.C. this week, where she is leading a group of a couple hundred people to ask the Food and Drug Administration to allow them access to a drug they say will save their children’s lives.

Christine McSherry’s son Jett has Duchenne Muscular Dystrophy, a progressive and debilitating disease that begins to affect boys at 6-7 years old, robbing them of mobility and motor function. It is terminal.

But four years ago, a new drug created by Cambridge’s Sarepta Pharmaceuticals was tested on twelve boys. Jett was on the drug for nine weeks, and began to show significant improvement.

“He’s able now to take a fork and bring it to his mouth,” said McSherry. “As a sophomore in college, that means the world to him.”

The problem is that the FDA hasn’t approved the drug yet, after four years of successful testing. That’s why McSherry and about 350 parents and supporters will be meeting with the FDA on Friday.

McSherry said the FDA is getting caught up in scientific methods and definitions, while the clock is ticking for the children affected.

“Boys shouldn’t have to wait,” said McSherry. “FDA needs to realize some of their outcome measures aren’t necessarily the most important measures to parents and patients living with the disease.”

McSherry said that, while the drug affects a sub-group of Duchenne patients, she said it’s important because it is one of the first examples of personalized medicine for rare diseases–so approval would open the floodgates for the development of other rare disease drugs.

WBZ NewsRadio 1030’s Karen Twomey reports

 

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