For Vertex, Can One Billion-Dollar Breakthrough Beget Another?

Technically, Mark Sleeper doesn't know for sure he didn't get the placebo, but nothing in his 27 years of fighting the deadly lung disease cystic fibrosis has made him feel as good as the pill he recently took in a clinical trial. "It was kind of mind-blowing," he says.

The energy he usually spent coughing and simply breathing was suddenly available for use in daily living. At first, he stayed up for two days. After 15 days on the treatment, his lung capacity, measured by the amount of air he can breathe out in a second, went from 70% of normal for a man his age to normal.

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