Vertex Pharmaceuticals revolutionized the treatment of cystic fibrosis. It also has unfinished business.
The Boston drugmaker’s medicines for the genetic disease — which damages the lungs and other organs — have transformed the lives of patients, helping them abandon other time-consuming treatments, shrug off what would have been dangerous respiratory infections, and just live breathing easier. The drugs, a number of so-called modulators approved starting in 2012, have been one of the great biotech success stories.
But the medications don’t work for some 10% of patients, depending on which CF-causing mutations they inherit. These patients have watched as their peers’ lives have changed, while they remain stuck using old medications to manage their symptoms and to slow the worsening of their illnesses.
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