She seems like any other 5-year-old, but Haylee Fiske's body can't keep up. She 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 Dr. David Weinstein, Director, Glycogen Storage Disease Program, University of Florida.
The only treatment is a strict meal schedule.
Dr. Weinstein is working on gene therapy. 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 this dog go from struggling to walk, to running. Experts say the injection works like a band-aid that covers up the genetic mutation.
"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, PhD, Children's National Medical Center, Washington, D.C.
Humans trials have begun and could eventually help people like Drew Bonner.
"How's life any fun if you're just going to be a downer? Why not just live it and be happy?" said Bonner.
For now, Drew takes on his obstacles with a smile and waits for medicine's next solution.
Dr. Weinstein says without treatment, dogs with Haylee's condition die within an hour the treated dog is now 2-and-a-half years old.
Although the muscular dystrophy trials are now underway, the gene therapy treatment for Haylee's condition isn't as far along. Researchers plan to test it in dogs for at least another year before entering human trials.
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