Estimated read time: 2-3 minutes
Ed Yeates ReportingSelected kids with spinal muscular atrophy are coming to Salt Lake to see if some new drugs might stop the progression of their disease. In between tests and treatments, some went skiing today in Park City.
Lauren and Claire Gibbs have spinal muscular atrophy, a genetic disease that gradually destroys the function of motor neurons and muscles in the limbs, neck and chest. Along with other kids, they've been traveling to and from Salt Lake over the past several months for some unique experiments at Primary Children's Hospital.
Out of the 60 families involved in the clinical trials, five from Utah, Kansas, Michigan and California are here this week for the next phase of their treatment.
Is it possible some new drugs, called H Stack Inhibitors, might give these kids more strength and mobility, perhaps even stop the progression of their disease? That's what neurologist Kathryn Swoboda and her colleagues are trying to find out.
Kathryn Swoboda M.D., Neurologist, PCMC: "All of us want to see these kids walk again. That would be an exciting thing. Now Lauren is a type three, so she walks but even to give her more strength and more endurance, more energy, so she doesn't fatigue."
Though it's still too early to draw any conclusions, Tim and Natalie Gibbs are seeing some changes in their daughters. For five-year old Claire, "I can open my locker, my two lockers. Pull-ups and jumping jacks that I wasn't able to do before in the water."
Claire can also slide herself out of a chair. For Lauren, “I’ve been able to walk up the stairs.”
Natalie Gibbs, Mom: “Before, they were very tired. By the end of the day they were very exhausted. And now we notice that they have little energy bursts going on more than they ever had before.”
The girls raced each other on the ski slopes, each winning some of the time. But the real victory will come if these new drugs, and others soon to come, really work.