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Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophy

This new gene therapy treatment has a Minnesota family optimistic
This new gene therapy treatment has a Minnesota family optimistic 02:22

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Colton Belluzzo was diagnosed with a form of muscular dystrophy when he was a baby.

The disease has weakened his muscles over time.

But he and his family now have good reason to be optimistic. 

"He's an outgoing little boy. Loves to play outside," said Morgan Belluzzo, Colton's mom. 

Colton Belluzzo also loves to fish, ride horses and even give his own weather reports on camera. 

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Colton Belluzzo WCCO

But when he was 7 months old, Colton Belluzzo was diagnosed with Duchenne muscular dystrophy — a rare condition that affects 1 in 5,000 children, mostly boys. It weakens muscles over time, and most people with DMD don't live past the age of 30. 

"When it first started, no idea. You didn't even think about it as a possibility," said Dan Belluzzo, Colton's dad. 

Last year, Colton Belluzzo got to the point where he had a hard time standing up. That's when his family learned that Dr. Peter Karachunski of M Health Fairview Masonic Children's Hospital had a groundbreaking treatment he wanted to try. 

"Yes, we are optimistic. And it's first such treatment in his class," said Karachunski. 

In December, doctors essentially placed a copy of the dystrophin gene into a virus, and then injected that virus into Colton Belluzzo's body. 

"This spreads around and gets into the muscle cells," said Karachunski. 

The virus isn't harmful, but the gene it's carrying is known to put the brakes on the progression of DMD.   

The hope is that this therapy slows down Colton Belluzzo's disease, buying time for him to receive other treatments in the future.

"We were terrified. It's scary," said Morgan Belluzzo. "They are injecting your child with a virus, essentially." 

But Colton Belluzzo's parents, Morgan and Dan, knew they didn't have many other options. 

And so far, the results have been eye-opening. In three months they've watched their son get stronger. He's able to go up and down the stairs on his own. And if Colton Belluzzo has his way, he'll be playing baseball in no time. 

"Just his energy levels are higher. He can do things. He's playing a lot more," said Dan Belluzzo.

"It's amazing, it's amazing that we were able to make this happen for our child," said Morgan Belluzzo.

Karachunski said there are many more muscular dystrophy treatments that are currently in clinical trials that he believes could help Colton Belluzzo in the future.

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