A promising new treatment for a rare muscle-wasting disease is now available at Studer Family Children’s Hospital at Ascension Sacred Heart Pensacola.
A promising new treatment for a rare muscle-wasting disease is now available at Studer Family Children’s Hospital at Ascension Sacred Heart Pensacola, providing hope for families who previously had little.
Karsten Price, 9, recently became the first pediatric patient in the region to receive Spinraza, the first treatment approved by the Food and Drug Administration to treat spinal muscular atrophy (SMA), a rare neuromuscular disorder that affects the motor nerve cells in the spinal cord. Over time, SMA robs people of physical strength, taking away their ability to walk, eat or breathe.
Karsten’s parents, Keith and Justina, became concerned when their toddler had no interest in crawling or walking.
“He would sit and play on the floor, but would fall over and couldn’t get back up,” Keith recalled. “After many doctors’ visits, a blood test confirmed he had SMA when he was 18-months-old.”
Like most kids his age, Karsten loves to play video games and aspires to be a social media personality. He uses a motorized wheelchair for mobility, a feeding tube to receive vital nutrients and a breathing machine to strengthen his weakened lungs.
Justina said they never lost hope that a treatment would come along. When Spinraza became available two years ago, their dreams were finally being realized. But their elation turned to disappointment when they learned that the nearest treatment site was more than five hours away. Since treatment has to be administered every four months via spinal injection for the rest of the person’s life, travelling can be a significant barrier to starting and maintaining treatment.
“It was so frustrating to have a treatment that you know can help your child gain strength and keep him healthy, but not have access to it,” Justina said. “We’ve always turned to Children’s Hospital for Karsten’s advanced medical needs, so we were excited when they made it available.”
Pediatrician-in-Chief Dr. Jason Foland said Children’s Hospital is excited about extending hope to more families by bringing this life-saving treatment to the region.
“In an environment specifically designed with a child’s unique needs in mind, the Children’s Hospital has the advanced imaging capabilities and specialized neurosurgeons who can safely administer treatment in more complex spine cases,” he said. “This really demonstrates what we’re all about – increasing access to specialized pediatric services.”
Keith said Karsten is an optimistic kid who doesn’t believe in giving up. “For the first time, he has to think about what he wants to achieve with improved strength,” Keith said.
He’s set simple goals: he wants to gain better head control, be able to roll in bed and have stronger lungs.
To learn more about this therapy at Children’s Hospital, call 850-416-1217.