Today was another big step forward in getting Kalydeco in my hands! My CF mutations are dF508/R117H-5T. Today the FDA had an advisory committee meeting to discuss the results of the clinical trial of Kalydeco in patients with the R117H mutation. The committee voted to approve it, 13-2! Now I have to wait and see if the FDA will decide to go along with the committee's recommendation, which it usually does. The FDA has until the end of the year to decide.
I was almost in tears listening to the testimonies of people with R117H who have been on the drug. They all said they can't imagine going back to the way they were before Kalydeco. They all said they can now plan their retirements. They all said it was a miracle. I can not believe I might be able to feel that difference soon - to not cough anymore, to not be so tired and achy by the end of the day, to be able to fully breath out of my nose and really smell things again! And to have the confidence that I will be here to help raise my grandchildren :) Crossing everything!
5 days ago