NORFOLK, Va. — A gene therapy treatment that doctors said is life-altering for patients with Duchenne Muscular Dystrophy was approved by the FDA in June.
Now, the parents of one 5-year-old in Norfolk said they have hope again as their child is treated in what doctors at Children's Hospital of The King's Daughters (CHKD) claimed is a "historic" event.
"Hi my name's Karson," 5-year-old Karson Blanchard introduced himself.
He is going into first grade, and he likes many things other kids his age like.
One of those things?
"Mac and Cheese," said Karson. "And chocolate."
He and his family have been in and out of hospital rooms for most of his life.
"In infancy, Karson didn't really hit his gross motor milestones," explained Karson's mother, Katy Blanchard.
She said doctors found out Karson had Duchenne Muscular Dystrophy, a genetic condition that results in progressive wasting of the muscles. It is considered rare. It is usually recognized in kids between ages three and six and often reduces their lifespans. Many patients suffer from heart and/or respiratory failure in their 20s and 30s because of the condition.
Karson has been getting some treatment for the condition, but Wednesday he was at CHKD in Norfolk to try a new gene therapy: Elevidys.
"It really opens a lot of doors for what-ifs that weren't possible before," said Katy. "Of course, we don't know what exactly it will look like, but now we can wonder whether he will have to get therapy every week forever, or maybe he'll be able to walk through high school or into adulthood, where that really wasn't a possibility before."
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Doctors said the treatment works by making a protein similar to the one missing in Karson's muscles.
Clinicians said it is given in a single does and while it cannot reverse muscle damage, they believe it could prevent future impairment. They said CHKD's the first hospital in Virginia to administer the therapy and plan to use the treatment for other patients in the future.
But the best part for Karson?
"Not having to get poked," Karson said with a smile.
The treatment's only given to patients between 4 and 5-years-old.
Karson turns six in a couple of days.
