Global prevalence of CFTR variants with respect to their responsiveness to elexacaftor-tezacaftor-ivacaftor
Expanding the label of ETI to rare responsive CFTR variants will make at least 91.5 % of pwCF eligible to this disease-modifying therapy.
Authors
- Marco Zampoli (ORCID: https://orcid.org/0000-0002-6899-6137)
- Anders Lindblad (ORCID: https://orcid.org/0000-0002-0287-1876)
- Elise Lammertyn (ORCID: https://orcid.org/0000-0002-1661-8687)
- Stephanie Y. Cheng (ORCID: https://orcid.org/0000-0003-2890-8451)
- Hector Gutierrez (ORCID: https://orcid.org/0000-0003-1373-0734)
- Milan Maçek (ORCID: https://orcid.org/0000-0002-5173-5280)
- Elizabeth A. Cromwell (ORCID: https://orcid.org/0000-0002-5106-0607)
- Jacqui G. van Rens
- S.B. Carr (ORCID: https://orcid.org/0000-0003-0580-2478)
- Annalisa Orenti (ORCID: https://orcid.org/0000-0002-0932-2757)
- Lutz Naehrlich (ORCID: https://orcid.org/0000-0002-7146-6997)
- A. Faro
- Anne L. Stephenson (ORCID: https://orcid.org/0000-0002-4529-5653)
- Anna Zolin (ORCID: https://orcid.org/0000-0001-9134-6442)
- Bulent Taner Karadag (ORCID: https://orcid.org/0000-0003-0605-8871)
- Catherine A. Byrnes (ORCID: https://orcid.org/0000-0002-3032-3172)
- Alexander Elbert (ORCID: https://orcid.org/0000-0002-2134-5118)
- Egil Bakkeheim (ORCID: https://orcid.org/0000-0002-9005-2204)
- Rasa Ruseckaite (ORCID: https://orcid.org/0000-0002-9078-2696)
- Pierre‐Régis Burgel (ORCID: https://orcid.org/0000-0003-0903-9828)
- Lvrf da Silva-Filho
Institutions
- Oslo University Hospital (NO)
- St. Michael's Hospital (CA)
- Inserm (FR)
- University of Cape Town (ZA)
- University of Milan (IT)
- Guy's and St Thomas' NHS Foundation Trust (GB)
- Justus-Liebig-Universität Gießen (DE)
- Université Paris Cité (FR)
- Charles University (CZ)
- Royal Brompton & Harefield NHS Foundation Trust (GB)
- Cystic Fibrosis Foundation (US)
- Harefield Hospital (GB)
- Cystic Fibrosis Canada (CA)
- University of Alabama at Birmingham (US)
- Hôpital Cochin (FR)
- Assistance Publique – Hôpitaux de Paris (FR)
- University Hospital in Motol (CZ)
- Drottning Silvias barn- och ungdomssjukhus (SE)
- Institut Cochin (FR)
- Starship Children's Health (NZ)
- Red Cross War Memorial Children's Hospital (ZA)
- Monash University (AU)
- Marmara University (TR)
Publication Details
- Journal
- Journal of Cystic Fibrosis
- Published
- 2025-10-18
- DOI
- https://doi.org/10.1016/j.jcf.2025.10.007
- Citations
- 7
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 10.63