On the outside, she's any other five-year-old, but on the inside, Haylee Fiske's body can't keep up. Haylee suffers from glycogen storage disease. Her liver can't release sugar fast enough.
"Their brains end up with hypoglycemia, and they can either end up developmentally delayed, or unfortunately, even dying," said David Weinstein, M.D., MSc.
The only treatment for Haylee's condition is a strict meal schedule. Doctor David Weinstein at the University of Florida is pioneering a treatment. After two injections of genes designed to restore a faulty enzyme, a dog with the same condition can now eat a normal diet.
"The disease is so mild now that we have not had to do any medical intervention," said Dr. Weinstein.
Another treatment in dogs brings hope to patients with muscular dystrophy. A new drug cocktail helped one dog go from struggling to walk to running. Experts say the injection works like a band-aid that covers up the genetic mutation linked to muscle weakness.
"It's almost as if you were reading a recipe, and instead of putting salt, your mutation said put in lots of hot pepper instead. Well, your cake wouldn't be so great. This band-aid lets it skip over that wrong pepper instruction," said Eric Hoffman, Ph.D., Children's National Medical Center.
Trials of the treatment have begun in humans and could eventually help people.
For more information: Ivanhoe Broadcast News2745 W. Fairbanks Ave.Winter Park, FL 32789http://www.ivanhoe.comJulie Marks, Supervising Producer of Prescription: Healthjmarks@ivanhoe.comDirect Line: (407) 691-1500Viewer Line: (407) 740-0789 ext. 579