PEMBROKE PARK, Fla. — There is a new game changer for children with a certain form of muscular dystrophy.
Now, a child who was destined to end up in a wheelchair with possible heart and respiratory problems has the chance to be able to run and play.
That dream is a reality thanks to an innovative gene therapy, and one South Florida hospital is leading the way.
Nine-year-old Ka’Lee loves to climb and run and play outdoors, but when he was just a toddler, his mother Carmen Stinnett knew something wasn’t quite right.
“I knew because I have four children and they always met their milestones,” she said. “So I knew something was different. I knew something was wrong.”
That mother’s intuition led to a frightening diagnosis.
Ka’Lee has duchenne muscular dystrophy, a potentially deadly genetic disorder that causes patients, as they get older, to become weaker and lose their ability to walk.
But now there is hope for children like Ka’Lee in the form of a new therapy that restores some of the genes these children are missing.
It’s called elevidys, a one-time infusion with big results.
“With gene therapy, you are trying to correct the underlying gene problem,” said Dr. Migvis Monduy, Nicklaus Children’s Hospital. “It’s not a cure for this particular disease, because the dystrophin gene is one of the bigger ones in the body. It’s too big to replace altogether.”
The gene therapy has been approved for children four years and older.
For Stinnett and Ka’Lee, a mother’s intuition, an early diagnosis, and innovative medical technology has been a winning combination.
“Before he didn’t run. He runs now,” Stinnett said. “He walks a lot better; he has more stability. It’s really helped him a lot.
“I am happy and blessed. I was so happy he got the gene therapy. I was worried and concerned, but this has been life changing.”
Nicklaus Children’s Hospital was one of the first hospitals in the country to offer the therapy.
Monduy also said that because early diagnosis is vital, starting in 2027 in Florida, newborns will be tested for duchenne muscular dystrophy in the hospital.
PEMBROKE PARK, Fla.
