Vertex Cystic Fibrosis Drug a Game-Changer

Vertex Pharmaceuticals (VRTX Quote) in the next week or so is expected to release new data on an experimental cystic fibrosis drug that could change the way the disease is treated.

VX-770 works by improving the function of a particular gene that is known to cause cystic fibrosis when defective. This is game-changing territory in cystic fibrosis research, because there are no drugs currently that address the underlying causes of the disease, only those that can treat the symptoms.

The downside to VX-770 is that it may only work for a very small percentage of cystic fibrosis patients who have this specific gene mutation. ...