“In venoms, nature has created a huge pharmacy of substances that block certain receptors and activate others.”

He was, by appearance, a healthy boy. He had a brilliant smile, striking blonde hair and eyes the color of the sea. He was fast becoming enamored with the stuff of boyhood: cars, sports and, especially, pirate ships.

But something wasn't right.

JB wasn't rolling over properly. He didn't crawl like other kids. Worried, his parents consulted with doctors and came home with painful news: Their son had Duchenne muscular dystrophy, a genetic disorder that causes a child's muscles to waste away.

The disease afflicts primarily boys, and the symptoms are chilling. Most victims are in wheelchairs by the time they're 12. Later, the heart and diaphragm deteriorate, making it difficult to breathe. Few patients survive beyond their 30s.

JB's story, however, is actually one of hope. Faced with a devastating diagnosis, his family joined the ranks of advocates hunting for treatments—for a way to save JB’s life.

In 2009, his grandfather, Jeff Harvey, founded a small pharmaceuticals firm with University at Buffalo scientists to develop a drug the researchers discovered in a peculiar place: the venom of a South American desert spider.