ORKAMBI is the first and only medicine to treat the underlying cause of CF for people with this mutation. D., Executive Vice President and Chief Medical Officer at Vertex."We believe it is important to treat the underlying cause of the disease as early as possible in these patients." The approval is based on data from a previously announced open-label Phase 3 clinical safety study of ORKAMBI presented at the 39 in the first half of 2017 for children ages 6 through 11 who have two copies of the F508del mutation.
(lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation.
People with this mutation represent the largest population of those with CF, a rare, life-threatening disease.
Her boyfriend, Ryan Denkenberger, 20 — the proverbial "friend of a friend" she met on Facebook — was at her side, watching a ventilator do her breathing as she lay on the bed, sedated. It was nothing fancy, he says, just dinner and a movie. a rock, whose mother raised him right.” He would help her carry her oxygen, and when she couldn’t make it up to the second-story of her home, Ryan would put her on his back and climb the stairs piggyback style.
Ryan was a young man in love, but he didn’t know if Ashley was going to survive. “I wanted her to know just how much I loved her, no matter what,” says Ryan, who works in the energy industry. “I thought it couldn’t get any better than that.” But it could, and did.
The investigators will conduct a study where all participants will receive Flumist® in the nose.