Expanding access to CFTR modulators for rare mutations: The utility of n-of-1 trials
Author:
Publisher
Elsevier BV
Subject
Pulmonary and Respiratory Medicine,Pediatrics, Perinatology and Child Health
Reference13 articles.
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1. Expanding the indication of CFTR modulator combinations for people with cystic fibrosis with non-F508del variants;The Lancet Respiratory Medicine;2024-08
2. Repeatability and reproducibility of the Forskolin-induced swelling (FIS) assay on intestinal organoids from people with Cystic Fibrosis;Journal of Cystic Fibrosis;2024-07
3. In silico analysis and theratyping of an ultra-rare CFTR genotype (W57G/A234D) in primary human rectal and nasal epithelial cells;iScience;2023-11
4. Managing cystic fibrosis in children aged 6-11yrs: a critical review of elexacaftor/tezacaftor/ivacaftor combination therapy;Expert Review of Respiratory Medicine;2023-02-01
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