M
Mommafirst
Guest
I went to a talk last week where Dr. Beall (the pres. of CFF) spoke. He discussed exactly what you are saying and it is very very exciting. One pill, once a day and anyone with those mutuations could have SIGNIFICANT help with their CF. Trials thus far are showing an average of 12% PFT increases in just the first 14 days!!!!
My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.
Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.
My daughter doesn't have either of the mutations that VX-770 and VX-809 help, but I believe that this is the first step toward a more global understanding how to make a huge difference in CF care.
Atalauren (for those mutations with an X at the end) is not doing as well in clinical trials. : ( For some reason it has had great outcomes in Israel trials, but not so great in the US. I hope that this one can yield better results -- my daughter has an X mutation.