Positive results from a study of ivacaftor (Kalydeco®) in children ages 1 to 2 were announced today.
Two Phase 3 clinical trials of tezacaftor (VX-661) in combination with ivacaftor (Kalydeco®) showed positive results, Vertex Pharmaceuticals announced today.
Before you can walk a mile in a person with CF's shoes, you have to put on a nebulizer.
When your life is already jam-packed with nebulizing medications, why would you add one more?
At first, it was easy to get my son to do his cystic fibrosis treatments. But as he got older, he started to hide his medications, rather than take them. I finally learned how to get him to see the importance of taking his medications.
Having already been underwhelmed by Orkambi® and Symdeko®, I was skeptical about whether the triple-combination therapy would live up to the hype. However, after a month of being on Trikafta®, I feel improvements in my lung function, weight, and overall energy.
I am both excited and scared that my son will be old enough to try Trikafta when he turns 12 later this year. In some ways, I can hardly wait to see how he will benefit. But I also know that he might experience side effects, and I don't want him to have any setbacks.
We depend on antibiotics to help us control and get rid of dangerous infections. But, in addition to the near-miraculous power to heal, many antibiotics come with serious side effects, too.
Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.