Hi ToriMom!
This drug has been a game changer for me. I am a heterozygous DF508. I tried Orkambi when it was new on the market and did not tolerate it at all. It was awful. When my physician offered this trial to me I knew I should try! I knew as soon as a couple days into the trial that I had to have received the drug and not the placebo. After being accepted into the Extended Access Program, it confirmed my initial thought. The biggest difference I have experienced is that I just, over all, feel good. I hadn't felt good in as long as I could remember. My PFTs have always been good; between 77% and 90% depending on whether or not I was fighting a bug, but have progressively been declining. The declines were between .25 and .50 percent every 6 months or so. Between the trial and starting the EAP, I have been on the medication going on 6 months. There has not been any decline in my PFTs, even with coming down the the flu (Influenza B) and common cold. The Influenza did not lead to Pneumonia, when before it most likely would have. I had a mile stone today- I ran about half a mile without feeling like I could keel over. I was so excited. With the medication I have noticed weight gain. Unlike a lot of CFers, I am already slightly overweight so that hasn't been too much welcomed by me. I am learning I need to watch what I eat, when before I was able to eat anything!
I hope your daughter can get on this medication and has great results! Feel free to reach out to me if you have any questions!
