“Impossible is just a big word thrown around by small men who find it easier to live in a world they’ve been given than to explore their power to change it. Impossible is not a fact. It’s an opinion. It’s a dare.”                              Muhammad Ali   

 March 20, 1998 – Walnut Creek, California: John and Aileen Crowley were on top of the world. Thirty-year-old John had graduated from Georgetown University and then received a Juris Doctor from Notre Dame Law School before earning a Master of Business Administration from Harvard Business School. He earned a nice salary with a top financial consulting firm in San Francisco. The young couple had two children, Megan, age 15 months, and Patrick, a week-old newborn.

Aileen had become concerned that Meghan wasn’t walking, but John reassured her that it was nothing. Their daughter was just a late walker. But to relieve Aileen’s concern, they took Megan to the doctor. After receiving the test results a week later, the doctor called the young parents to his office. “I am very sorry,” he began, “but Megan has a rare genetic disorder called Pompe Disease. It is a severe neuromuscular disorder, and most children die from respiratory failure or cardiac arrest. The life expectancy is two years. There is no cure. You should enjoy the time that you have with her.” The following week, tests revealed that Patrick also had the disease.

Several months later, Megan almost died from pneumonia. John promised her that he would find a special medicine to help her. In July 1998, he quit his job and moved the family to Princeton, New Jersey, where he joined Bristol-Myers Squibb, a large pharmaceutical company that was researching cures for diseases like Pompe. As the new marketing director in the research division, John could learn more about genetic disorders.

A year later, John discovered that Novazyme, a small four-person company in Oklahoma City, Oklahoma, focused exclusively on Pompe research. He convinced a venture capital group to invest $8 million in the company, then quit his job with Bristol-Myers and joined Novazyme. John was determined that the research would be successful; his children’s lives depended on it.

By 2001, Novazyme had not developed a cure for Pompe. John, now the company CEO in Boston, had quickly grown the company and sold it for $225 million to Genzyme, another biotech enterprise stipulating that he would become Genzyme’s senior VP and lead their Pompe research as part of the deal.

By this time, Megan and Patrick were wheelchair-bound and dependent on ventilators to breathe. Their hearts were twice the normal size. Time was running out. Doctors predicted the children could not possibly live beyond age five, and Megan would be five in a month. John’s urgency increased, and he drove the research team relentlessly as the company evaluated four enzymes that might provide a cure.

On a Friday night in October 2002, John received a call that Megan and Patrick had been accepted to a clinical trial involving 12 children using one of the Genzyme drugs. In early January, John and Aileen pushed the button to start the children’s enzyme drip at St. Peters University Hospital in New Brunswick, New Jersey. The children received the drug every other week.

At the first checkup in April, both children’s hearts had reduced to normal size, and their neck and shoulder muscles had grown marginally stronger. Genzyme’s $200 million investment in a special medicine, myozyme, appeared to be helping control the symptoms of Pompe for Megan, Patrick and hundreds of other children worldwide.

Today, 26-year-old Megan and 25-year-old John live at home with John and Aileen. They are wheelchair-bound and supported by a round-the-clock nurse. On March 13, 1998, John’s life and career trajectory changed when Megan was diagnosed with Pompe.

More than two decades after he quit his job to find a special medicine for Megan, he is the CEO of Amicus Therapeutics, a global biotech company that he started in 2005.  He is also the chief executive of Biotechnology Innovation Organization, a trade group comprised of small health-focused medical and pharmaceutical companies.

John, 56, is frequently in Washington, D.C., as he lobbies the Food and Drug Administration to spend more money for genetic disease research. He saved his children’s lives and now he is chasing another miracle. He wants to find a cure for their disease.