Article
Theratyping cystic fibrosis patients to guide elexacaftor/tezacaftor/ivacaftor out-of-label prescription.
The European respiratory journal - 1 Oct 2023
Dreano Elise, Burgel Pierre Régis, Hatton Aurelie, Bouazza Naim, Chevalier Benoit, Macey Julie, Leroy Sylvie, Durieu Isabelle, Weiss Laurence, Grenet Dominique, Stremler Nathalie, Ohlmann Camille, Reix Philippe, Porzio Michele, Roux Claude Pauline, Rémus Natacha, Douvry Benoit, Montcouquiol Sylvie, Cosson Laure, Mankikian Julie, Languepin Jeanne, Houdouin Veronique, Le Clainche Laurence, Guillaumot Anne, Pouradier Delphine, Tissot Adrien, Priou Pascaline, Mély Laurent, Chedevergne Frederique, Lebourgeois Muriel, Lebihan Jean, Martin Clémence, Zavala Flora, Da Silva Jennifer, Lemonnier Lydie, Kelly-Aubert Mairead, Golec Anita, Foucaud Pierre, Marguet Christophe, Edelman Aleksander, Hinzpeter Alexandre, de Carli Paola, Girodon Emmanuelle, Sermet-Gaudelus Isabelle, Pranke Iwona
Abstract excerpt
BACKGROUND: Around 20% of people with cystic fibrosis (pwCF) do not have access to the triple combination elexacaftor/tezacaftor/ivacaftor (ETI) in Europe because they do not carry the F508del allele on the CF transmembrane conductance regulator (CFTR) gene. Considering that pwCF carrying rare variants may benefit from ETI, including variants already validated by the US Food and Drug Administration (FDA), a...
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