Article
Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor.
BMJ open respiratory research - 1 May 2023
Higgins Mark, Farietta Thalia, Campbell Daniel, Liu Meng, Ostrenga Josh, Elbert Alexander, Shih Judy, Volkova Nataliya
Abstract excerpt
BACKGROUND: Ivacaftor approval was extended to people with cystic fibrosis (CF) and an R117H variant in 2014 in the USA. This observational, real-world, postapproval study evaluated long-term outcomes among people with CF and an R117H variant on ivacaftor using data from the US Cystic Fibrosis Foundation Patient Registry. METHODS: Key outcomes were evaluated in ivacaftor-treated people with CF and an R117H...
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