Cristina Might drew close to her son. He was listless and groggy after weeks of battling a puzzling illness that had filled his lungs with fluid and, hours earlier, stopped his breathing entirely. A code team had rushed to Buddy's bedside and jolted him back to life, but now the 11-year-old with the broad smile was gray, his eyes unable to focus. His mom leaned nearer still. It was time to say goodbye. But Cristina's words to her son, a brown-eyed boy who loved dolphins and his aquarium, offered no hint of her desperation: "I was telling him it was all going to be OK, that his fishies couldn't wait to see him again and that he had to hurry up and come home." Somehow, Buddy made it through that night this past May, allowing doctors at Children's Hospital of Alabama to insert a tube to drain his lungs. His illness had caused a frightening cascade of symptoms: a yellowish substance in his bones and a bulging abdomen, on top of the deluge of fluid.

Human biology is full of surprises -- especially for drug makers. Viagra wasn't designed for erectile dysfunction. Both drugs were meant to treat cardiovascular issues (as sildenafil and minoxidil, respectively), until patients reported their sexual and follicular side effects. When his son was diagnosed with an ultra-rare disease, computer scientist Matt Might, Ph.D., kicked off a search for answers. His quest led to partnerships with researchers across the country, a White House appointment, a faculty position at Harvard, and a profile in the New Yorker.