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Article

Who are the 10%? - Non eligibility of cystic fibrosis (CF) patients for highly effective modulator therapies.

Respiratory medicine - 1 Aug 2022

Desai Maya, Hine Chris, Whitehouse Joanna L, Brownlee Keith, Charman Susan C, Nagakumar Prasad

Abstract excerpt

BACKGROUND: The availability of mutation-specific cystic fibrosis modulator therapies has the potential to improve the lives of children and adults with cystic fibrosis. The frequency of mutations causing defects in the cystic fibrosis transmembrane conductance regulator (CFTR) function varies between sub-groups in multi-ethnic populations. The profile of patients eligible for CFTR modulator...

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Identifiers

PMID: 35633605
DOI: 10.1016/j.rmed.2022.106878

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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