‘We’re Still Waiting’: As Cystic Fibrosis Drugs Deliver New Hope, Not Everyone Is Being Swept Up by Scientific Progress

February 4, 2019

(STAT News) – The catch is that cystic fibrosis is not caused by one mutation, or a handful, but more than 1,500 different rearrangements in the code for the gene known as CFTR. The cutting-edge treatments — there are three available now and a fourth, still-experimental medication expected to be approved, all from Vertex Pharmaceuticals — cover the mutations held by some 90 percent of CF patients. That leaves up to 10 percent of people whose diseases are advancing without a powerful defense to slow them down. Hillman is among them.

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