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The expanded French compassionate programme for elexacaftor–tezacaftor–ivacaftor use in people with cystic fibrosis without a F508del CFTR variant: a real-world study

Pierre‐Régis Burgel, Isabelle Sermet‐Gaudelus, Emmanuelle Girodon, I. Durieu, Véronique Houdouin, Camille Audousset, Julie Macey, Dominique Grenet, Michele Porzio, M. Murris‐Espin,

The Lancet Respiratory Medicine · 2024 · 47 citations · 30 references

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30

Multi-ethnic reference values for spirometry for the 3–95-yr age range: the global lung function 2012 equations

Philip H. Quanjer, Sanja Stanojevic, Tim Cole et al. · European Respiratory Journal · 2012 · 5.9K citations · Full text

, , +12

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Peter G. Middleton, Marcus Mall, Pavel Dřevı́nek et al. · New England Journal of Medicine · 2019 · 2.1K citations · Full text

, , +16

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

Harry Heijerman, Edward F. McKone, D.G. Downey et al. · The Lancet · 2019 · 1.4K citations · Full text

, , +14

Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

Patrick R. Sosnay, Karen R. Siklosi, Fredrick Van Goor et al. · Nature Genetics · 2013 · 625 citations · Full text

, , +11

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

Radu G. Avramescu, Annette Chiang, Scott A. Houck et al. · Molecular Biology of the Cell · 2016 · 590 citations · Full text

, , +17