Article
CFTR modulator therapies - Effect on life expectancy in people with cystic fibrosis.
Paediatric respiratory reviews - 1 Jun 2022
Balfour-Lynn I M, King J A
Abstract excerpt
CFTR modulators have dramatically changed the clinical course of CF in those fortunate enough to receive them. Inevitably, randomised controlled trials during the development of these drugs are too short to use mortality as an outcome. Evidence for their effect on life expectancy are best gained from real world registry studies specifically looking at mortality, but these are only available for ivacaftor to date....
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
