The uneven advance of precision medicine

There’s been a renaissance in cystic fibrosis drugs, led by Vertex Pharmaceuticals’ ground-breaking drug, Kalydeco. These medications work in patients who have certain mutations in the CFTR gene. For some 90 percent of CF patients, these treatment can make a massive difference in their ability to breathe.

But the remaining 10 percent has not been as fortunate. And, overall, the new therapeutic landscape for CF has created disparities among patients who, until 2012, were all treated the same way, STAT’s Andrew Joseph writes.

“I worry about the perception that CF is done,” said 34-year-old Emily Kramer-Golinkoff, who is contemplating a lung transplant thanks to the lack of therapeutic options available. “There is an outlying 10 percent of us for whom it is not at all done.”