Children keep dying from mysterious illnesses that have been traced to tiny structures called mitochondria, and diagnosed with Mitochondria Disease.

If the term is unfamiliar, that's no surprise. "When I began working on mitochondrial disease back in the '80s," says Shoffner, "people were still arguing over whether it even existed." Nobody is arguing about that anymore. In fact, doctors have now identified hundreds of different subtypes of the disorder. What they all have in common is a malfunction of the mitochondria--tiny substructures, or organelles, found inside every cell in the body. Their job is to convert food into a chemical called ATP that cells use for energy. When they go bad, all sorts of havoc is wreaked on the body. Depending on which types of cells are affected, mitochondrial disease can cause muscle wasting, nerve damage, seizures, stroke, blindness, deafness and more.