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Ed Yeates reportingA national support group calls it "Project Cure,"and it may be the first step in treating children with spinal muscular atrophy.
Kevin and Bonnie Shiesley from western New York, are in Salt Lake City with their two year old son,
hoping a new drug might stop the progression of his disease.
Bonnie Shiesley, Kale's Mother: "ABOUT 15 MONTHS OF AGE, HE STOPPED BEARING WEIGHT ON HIS LEGS AND WE WEREN'T QUITE SURE WHAT IT WAS."
In Buffalo, two months later, Kale was diagnosed with Spinal Muscular Atrophy, a genetic disease that gradually destroys the function of motor neurons - and muscles in the limbs, neck and chest.
KEVIN SHIESLEY / KALE'S FATHER: "AND THEN ALL OF A SUDDEN, IT WAS BOOM. AND WE WERE SHOCKED BECAUSE THE NEUROLOGIST WALKED IN AND GAVE US THIS DIAGNOSIS AND THEN WALKED OUT OF THE ROOM."
But it's a lot different today - in this room - at Primary Children's Hospital. Neurologist Kathryn Swoboda and therapist Janine Wood are measuring what Kale can or can't do - in preparation for an experiment. He's about to get the FIRST EVER drug which could restore the missing chemical messages blocked by SMA.
ED YEATES, SCIENCE SPECIALIST: "KALE WILL BE TESTED ON WHAT IS CALLED VALPROIC ACID. IT'S HAD OTHER APPLICATIONS BUT IT'S NEVER BEEN USED BEFORE IN THE TREATMENT OF SMA."
KATHRYN SWOBODA, NEUROLOGIST, PCMC: "EVEN IF WE COULD STABILIZE THESE KIDS, PARTICULARLY IF WE COULD START THE DRUG AT AN EARLY AGE, THEN THAT WOULD MAKE ME REALLY HAPPY. BUT I THINK THE BEST THING WOULD BE OBVIOUSLY IF WE COULD GET THEM STRONG ENOUGH TO WALK AGAIN. AND THAT'S WHAT EVERYONE'S HOPE IS."
As the Shiesley's travel to and from Salt Lake in the months to come - everybody will be watching how, if at all, Kale's mobility changes. While he's too young to know what this is all about - he's making a lot of new friends here.
"THANK YOU. THANK YOU"
Primary Children's will also be testing a second new drug on children younger than Kale with more severe forms of SMA.
Families of SMA is a nonprofit organization based in Libertyville, IL. FSMA has funded $12 million towards SMA research, and has already committed an additional $10 million over the next three years. For more information, follow the link to the web site or call 1-800-886-1762.