Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rare<i>CFTR</i>variants: a viewpoint-Reference-Cited by-同舟云学术

Gathering real-world compassionate data to expand eligibility for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with N1303K or other rareCFTRvariants: a viewpoint

Published:2024-01 Issue:1 Volume:63 Page:2301959
ISSN:0903-1936
Container-title:European Respiratory Journal
language:en
Short-container-title:Eur Respir J

Author:

Burgel Pierre-Régis^ORCID,Sermet-Gaudelus Isabelle^ORCID,Girodon Emmanuelle^ORCID,Kanaan Reem,Le Bihan Jean,Remus Natascha,Ravoninjatovo Bruno,Grenet Dominique,Porzio Michele,Houdouin Véronique,Le Clainche-Viala Laurence,Durieu Isabelle,Nove-Josserand Raphaele,Languepin Jeanne,Coltey Bérangère,Guillaumot Anne^ORCID,Audousset Camille,Chiron Raphaël,Weiss Laurence,Fajac Isabelle,Da Silva Jennifer,Martin Clémence^ORCID

Funder

Filière Maladies Rares Muco CFTR

Société Française de la Mucoviscidose

Association Vaincre la Mucoviscidose

Publisher

European Respiratory Society (ERS)

Subject

Pulmonary and Respiratory Medicine

Reference32 articles.

1. Molecular structures reveal synergistic rescue of Δ508 CFTR by Trikafta modulators;Fiedorczuk;Science (New York, NY),2022

2. 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

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

4. Safety and efficacy of elexacaftor/tezacaftor/ivacaftor for 24 weeks or longer in people with cystic fibrosis and one or more F508del alleles: interim results of an open-label phase 3 clinical trial;Griese;Am J Respir Crit Care Med,2021

5. Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: interim results of a long-term registry-based study;Bower;J Cyst Fibros,2023