You should note, though, that PTC 124 will only "fix" CF genes that have a "nonsense mutation." That affects about 10% of CFers. As a general rule, mutations that end in X are nonsense mutations, but there are others. Check the PTC website for more info. Right now they are in the planning phase for a second round of phase 2 trials. After that, they'd have to do phase 3 trials before it can be marketed (Assuming it's successful). The results so far have been promising -- my heart expands when I think my daughter may be "cured" of her symptoms with just a pill!