RIVES JUNCTION, Mich. (WLNS) – Muscular dystrophy impacts the lives of millions of people.
One type called Duchenne can be devastating for young people and their families.
But thanks to a new drug, hope could be on the horizon.
6 News talked to a family from Jackson County that’s taking advantage of a new treatment.
When Brandon Martz was a toddler, his parents realized he had a tough fight ahead of him.
“They did an evaluation and he was diagnosed with development delay for a year. And then he was diagnosed with muscular dystrophy,” said Brandon’s mother, Cinni Martz.
Brandon has Duchenne muscular dystrophy.
It’s a type of MD that only occurs in boys.
“It’s fatal. It’s a terminal disease,” Cinni said.
Brandon has beaten the odds.
He’s been able to reach his teen years and function independently.
And it’s all thanks to a new drug.
“Three times a day he takes it. It’s a powder that we mix for him,” Cinni said.
The drug is called Translarna.
“It creates a gene that can work in some fashion to keep him stronger, said Brandon’s father, Doug Martz.
It’s allowed in 20 countries around the world.
But this drug is not yet approved by the FDA.
The Martz family was able to get it, because Brandon is taking part in a scientific study.
“Why is it that the FDA is standing so firm and not allowing us to use this drug and open it up? Brandon is using it and we’re seeing the benefits. We have seen no side effects. Only progress,” Doug said.
Boys with this condition often don’t make it into adulthood.
The Martz family hopes the FDA can allow the drug to be widely available in the U.S. so other families can have the same opportunity.
“My heart goes out, and her heart goes out to all the parents that we know that can benefit from this and don’t get a chance,” Doug said.
Brandon is a senior in high school.
And just like any other parents, Doug and Cinni want the best for his future.
“He’s talked a lot about college. Whatever makes him feel successful and happy,” Cinni said.