New drug treatment helps Livingston boy continue to walk – Billings Gazette

The particular form of muscular dystrophy that afflicts 6-year-old Trayton Backer is the result of busted proteins in the longest gene of his X chromosome.

In Montana, it makes Trayton one of kind.

Nationwide, only 13 percent of people withmuscular dystrophy have Duchenne's Trayton's diagnosis. And of those, Trayton is the only one in the state.

A new treatment, approved late last year by the U.S. Food and Drug Administration, could drastically improve Trayton's life and make his form of muscular dystrophy more manageable.

"This treatment really is a breakthrough,"said Trayton's doctor, John Binder, a pediatric neurologist at Billings Clinic.

Trayton was diagnosed when he was 4.

"I cried," said Emily Oldaugh, Trayton's mom. "The first thing you think of is, 'Oh, my kid's gonna be in a wheelchair.'"

Oldaugh had know for a couple of years that something wasn't right. Trayton walked late, his calves were larger than normal and he had trouble crawling up and down stairs.

As soon as the doctors were sure it was Duchenne muscular dystrophy, they began to treat it as best they could, she said.

But the broken gene at the heart of Duchenne muscular dystrophy has made it frustratingly difficult to treat. The disease progressively deteriorates and weakens the muscles in the body, starting at a young age in the lower limbs.

"These kids are in a wheelchair by age 10," Binder said.

When working properly, the gene programs the muscle protein, dystrophin. The lack of thedystrophin protein in Trayton's muscles is what causesthem to deteriorate.

And that's what's so exciting about the new treatment, Binder said. It's essentially a patch on that broken gene, allowing it to create the dystrophin protein.

"This is the first treatment that's actually working at the level of the genetic abnormality," said Binder."It's not a cure but it'll make a major difference."

It's cutting edge stuff. And, it's expensive. Oldaugh and Trayton had to travel from their home in Livingston to Billings once a week for the hour-long infusion that administered the medicine.

Each infusion costs roughly $11,000. When Oldaugh first started working with Medicaid to get approval for the treatments last year, officials there had yet to hear of the medicine.

Eventually it was approved and Medicaid agree to cover a portion of the infusions. What costs were left are being covered through Billings Clinic's medication assistance program.

With four treatments under his belt, Trayton can now start receiving the infusions at home. No more weekly trips to Billings.

"He'll miss going out to lunch," Oldaugh said with a laugh.

Even though the treatment patches up the broken parts of the gene, it doesn't repair it. That means at some point, years down the road, the muscular dystrophy takes over and the muscles continue their slow deterioration.

Until then, the new treatment, along with regular physical therapy, will give Trayton more of a normal life than he'd otherwise have with traditional muscular dystrophy treatments.

"We'll add years of walking to his life," Binder said.

That is great for Trayton, who's getting ready for his big move from kindergarten to first grade at the end of the summer.

"He's a pretty normal kid," Oldaugh said, smiling.

He's active and loves playing with his friends, who know Trayton isn't exactly like they are.

"Kids are pretty smart," Oldaugh said.

But it doesn't impede their friendship and school has been one of Trayton's favorite things.

Moving forward, Oldaugh is grateful the treatment has bought her son more time but she knows at some point the disease will take its toll.

"We just try to stay really positive," she said.

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New drug treatment helps Livingston boy continue to walk - Billings Gazette