ORLANDO, FLA. (WSVN) - Groundbreaking gene therapy provided by a hospital in Central Florida changed the life of a little boy.
The 4-year-old suffers from Duchenne Muscular Dystrophy or DMD, which is a disease that causes progressive loss of muscle mass.
He received a one time treatment called Elevidy’s at Nemours Children’s Hospital in Orlando.
“This is the first time where we’re able to actually treat the underlying cause of Duchenne Muscular Dystrophy. We’re able to replace the missing gene in a substantive way,” said Dr. Omer Abdul Hamid, a pediatric neurologist at Nemours Children’s hospital. “It’s really been a game-changer for our families and the community as a whole.”
“I’ve been looking forward to getting Jaxson’s gene therapy and I am just grateful that its here,” said Angela Ramirez, the boy’s mother.
According to experts, an estimated 20,000 children are diagnosed with Duchenne globally each year. With the addition of this new drug, it may help stop the disease all together.
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