Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programme

Authors

Karen S. Raraigh (ORCID: https://orcid.org/0000-0001-6730-6854)
Isabelle Sermet‐Gaudelus (ORCID: https://orcid.org/0000-0001-5537-9482)
Souphatta Sasorith (ORCID: https://orcid.org/0000-0001-8448-1726)
Jennifer Da Silva (ORCID: https://orcid.org/0000-0001-6897-3616)
Emmanuelle Girodon (ORCID: https://orcid.org/0000-0001-7913-4682)
Clémence Martin (ORCID: https://orcid.org/0000-0003-4385-5888)
Caroline Raynal (ORCID: https://orcid.org/0000-0002-1070-0583)
Pierre‐Régis Burgel (ORCID: https://orcid.org/0000-0003-0903-9828)
Neeraj Sharma (ORCID: https://orcid.org/0000-0001-8474-3714)

Institutions

Hôpital Necker-Enfants Malades (FR)
Centre National de la Recherche Scientifique (FR)
Johns Hopkins University (US)
Inserm (FR)
Université Paris Cité (FR)
Johns Hopkins Medicine (US)
Physiologie et Médecine Expérimentale du Coeur et des Muscles (FR)
Hôpital Cochin (FR)
Assistance Publique – Hôpitaux de Paris (FR)
Institut de Génétique Moléculaire de Montpellier (FR)
Institut Cochin (FR)

Publication Details

Journal: EClinicalMedicine
Published: 2025-08-30
DOI: https://doi.org/10.1016/j.eclinm.2025.103476
Citations: 8
Primary Topic: Cystic Fibrosis Research Advances
Type: article
Field-Weighted Citation Impact: 12.15

Funders

Association Vaincre la Mucoviscidose

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article

Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programme

Karen S. Raraigh, Isabelle Sermet‐Gaudelus, Souphatta Sasorith, Jennifer Da Silva et al.

Aug 30, 2025

8 citations

EClinicalMedicine

Cystic Fibrosis Research Advances

12.15

article

Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programme

Karen S. Raraigh, Isabelle Sermet‐Gaudelus, Souphatta Sasorith, Jennifer Da Silva, Emmanuelle Girodon, Clémence Martin, Caroline Raynal, Pierre‐Régis Burgel, Neeraj Sharma

August 30, 2025article en

8 citations

Abstract

No abstract available for this paper.

EClinicalMedicineVol. 88

Hôpital Necker-Enfants Malades (FR), Centre National de la Recherche Scientifique (FR), Johns Hopkins University (US), Inserm (FR), Université Paris Cité (FR), Johns Hopkins Medicine (US), Physiologie et Médecine Expérimentale du Coeur et des Muscles (FR), Hôpital Cochin (FR), Assistance Publique – Hôpitaux de Paris (FR), Institut de Génétique Moléculaire de Montpellier (FR), Institut Cochin (FR)

Association Vaincre la Mucoviscidose

Openalex Percentile: Top 1%

Cystic Fibrosis Research Advances

12.15

DOI Open Access PDF OpenAlex

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Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR Class I variants: real-world data from the French compassionate programme — Karen S. Raraigh, Isabelle Sermet‐Gaudelus, et al. · EClinicalMedicine (2025) | TGRS Research Map | TGRS