‘Life-Saving’ Drug Gives New Life to Kids Fighting Rare Form of Muscular Dystrophy

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SACRAMENTO --

At first glance, 9-year-old Gavin Carlone seems like any other child. He loves football and candy. You’d never guess that this little boy is in the fight of his life.

Gavin’s mother, Tonya Carlone, has been by his side as he's endured many medical tests while battling a rare form of muscular dystrophy.

“This is a very progressive, fatal muscle disease with no cures and no therapies, and we are hoping the FDA approves this life-saving medication,” Tonya Carlone said.

A disease known as Duchenne muscular dystrophy has been attacking the muscles in Gavin's body since he was a young child. It’s so severe it can rob him of the freedom of movement and possibly his life.

Clinical researcher and medical professional at UC Davis Medical Center Erica Goude has been treating Gavin for years. She described his specific disease.

“They start declining more and that’s where they start having respiratory and cardiac failure, and they usually pass away in their 30s,” Goude said.

Once a month, the family travels from Seattle to UC Davis Medical Center in Sacramento for a specific treatment. A single shot of medicine administered to Gavin’s arm helps make his life easier, and the disease a bit more bearable. It’s at UC Davis Med Center that the family has found hope.

The UC Davis medical facility is one of the only research labs on the West Coast that can administer and study an experimental drug called drisapersen.

Professor of physical medicine Craig McDonald has been leading the treatment for Gavin and many others.

“I’ve been treating young men and boys with Duchenne Muscular Dystrophy for 25 years, and I never dreamed we would see this type of therapy available to them, which could actually stabilize this horrible disease progression,” McDonald said.

For McDonald, the drug is a game changer.

“It’s incredibly rewarding and gratifying to be able to see someone like Gavin and other patients have this kind of treatment effect,”he said.

For Gavin, the medication has changed his life.

“Today, after being on drisapersen, an amazing medication, my son is riding a bike, he’s running, he’s playing on a soccer team for the first time,” Tonya Carlone said.

While the drug hasn’t been approved by the FDA, doctors hope children like Gavin will continue to benefit as the medication gives them something money can’t buy -- time.