The effectiveness of CFTR modulators in people with CF and rare mutations: A real-world study

Pediatr Pulmonol. 2024 Jan;59(1):221-224. doi: 10.1002/ppul.26713. Epub 2023 Oct 11.

Authors

Sofia Hanger¹, Imogen Felton¹, Emem-Fong Ukor¹, Elaine Bowman¹, Clare Caldwell¹, Winston Banya², Susan Madge¹, Andrew L Jones¹, Nicholas J Simmonds^{1

3}

Affiliations

¹ Adult Cystic Fibrosis Centre, Royal Brompton Hospital, London, UK.
² Medical Statistics, Research Office, Royal Brompton Hospital, London, UK.
³ National Heart and Lung Institute, Imperial College London, London, UK.

PMID: 37817743
DOI: 10.1002/ppul.26713

No abstract available

Publication types

Letter

MeSH terms

Cystic Fibrosis Transmembrane Conductance Regulator* / drug effects
Cystic Fibrosis* / drug therapy
Cystic Fibrosis* / genetics
Humans
Mutation

Substances

CFTR protein, human
Cystic Fibrosis Transmembrane Conductance Regulator