Experiences with

Ivacaftor

The treatment, which combines elexacaftor, tezacaftor and ivacaftor, is now available on the NHS bringing hope to thousands of people living with CF across the UK. https://news.liverpool.ac.uk/2021/01/11/benefits-of-transformational-cystic-fibrosis-drug-highlighted-in-new-review/ Cochrane Reviews:

Lurie Children’s Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del mutation in the CFTR gene, which is estimated to represent almost 90

The rate of discontinuation was considerably higher than previously reported in clinical trials, the authors wrote. https://www.newswise.com/articles/cf-patients-experience-improved-lung-health-with-lumacaftor-ivacaftor-but-with-caveats