It truly is remarkable, not only for the vast improvement in outcome for patients, but also the science. The genetic defect that causes CF results in a chlorid

It truly is remarkable, not only for the vast improvement in outcome for patients, but also the science. The genetic defect that causes CF results in a chloride channel receptor being malformed so it it doesn't function correctly. The fact they were able to develop a small molecule that perfectly fits within the defective receptor and then causes the receptor to change conformation so that it's now functional is absolutely remarkable. Then layer on top the company has developed a few molecules that can address the different defects in different patients is really amazing science. It's transformed a disease where patients die in their 30's or undergo a risky lung transplant (and still suffer other aspects of the disease after) to a more moderate, treatable disease where life span is normal is one of the great medical achievements of our lifetime.