Real‐life efficacy and safety of elexacaftor/tezacaftor/ivacaftor on severe cystic fibrosis lung disease patients

Author:

Kos Renate1ORCID,Neerincx Anne H.1,Fenn Dominic W.12,Brinkman Paul1,Lub Rianne1,Vonk Steffie E. M.3,Roukema Jolt4,Reijers Monique H.5,Terheggen‐Lagro Suzanne W. J.6,Altenburg Josje1,Majoor Christof J.1,Bos Lieuwe D.17,Haarman Eric G.6,Maitland‐van der Zee Anke H.16,

Affiliation:

1. Department of Respiratory Medicine Amsterdam University Medical Centres – loc. AMC Amsterdam The Netherlands

2. Laboratory of Experimental Intensive Care and Anaesthesiology Amsterdam University Medical Centres – loc. AMC Amsterdam The Netherlands

3. Department of Hospital Pharmacy Amsterdam University Medical Centres – loc. AMC Amsterdam The Netherlands

4. Department of Paediatric Pulmonology, Amalia Children's Hospital Radboud University Medical Center Nijmegen The Netherlands

5. Department of Pulmonology Radboud University Medical Center Nijmegen The Netherlands

6. Department Paediatric Respiratory Medicine and Allergy, Emma Children's Hospital Amsterdam University Medical Centres Amsterdam The Netherlands

7. Department of Intensive Care Amsterdam University Medical Centres – loc. AMC Amsterdam The Netherlands

Publisher

Wiley

Subject

General Pharmacology, Toxicology and Pharmaceutics,Neurology

Reference18 articles.

1. Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

2. Cystic Fibrosis Foundation Patient Registry 2019 Annual Data Report Bethesda Maryland Maryland.2020.https://www.cff.org/Research/Researcher‐Resources/Patient‐Registry/2019‐Patient‐Registry‐Annual‐Data‐Report.pdf

3. Nederlandse CF Registratie 2020. Baarn.2020.

4. 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

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

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