Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2–5 Years with Cystic Fibrosis and at Least One F508del Allele

Author:

Goralski Jennifer L.1,Hoppe Jordana E.2,Mall Marcus A.345ORCID,McColley Susanna A.6,McKone Edward7ORCID,Ramsey Bonnie89,Rayment Jonathan H.10,Robinson Phil1112,Stehling Florian13,Taylor-Cousar Jennifer L.14ORCID,Tullis Elizabeth15,Ahluwalia Neil16,Chin Anna16,Chu Chenghao16,Lu Mengdi16,Niu Tao16,Weinstock Tanya16,Ratjen Felix17,Rosenfeld Margaret89ORCID

Affiliation:

1. University of North Carolina School of Medicine, Chapel Hill, North Carolina;

2. University of Colorado School of Medicine and Children’s Hospital Colorado, Aurora, Colorado;

3. Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité–Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany;

4. Berlin Institute of Health, Charité–Universitätsmedizin Berlin, Berlin, Germany;

5. German Center for Lung Research, Associated Partner, Berlin, Germany;

6. Ann & Robert H. Lurie Children’s Hospital of Chicago and Northwestern University Feinberg School of Medicine, Chicago, Illinois;

7. St. Vincent’s Hospital, Dublin, Ireland;

8. Seattle Children’s Research Institute, Seattle, Washington;

9. Department of Pediatrics, University of Washington School of Medicine, Seattle, Washington;

10. University of British Columbia and British Columbia Children’s Hospital, Vancouver, British Columbia, Canada;

11. The Royal Children’s Hospital, Murdoch Children’s Research Institute, Melbourne, Victoria, Australia;

12. Department of Pediatrics, University of Melbourne, Melbourne, Victoria, Australia;

13. Children’s Hospital, University of Duisburg-Essen, Essen, Germany;

14. National Jewish Health, Denver, Colorado;

15. St. Michael’s Hospital, University of Toronto, Toronto, Ontario, Canada;

16. Vertex Pharmaceuticals Inc., Boston, Massachusetts; and

17. University of Toronto, Toronto, Ontario, Canada

Funder

Vertex Pharmaceuticals

Publisher

American Thoracic Society

Subject

Critical Care and Intensive Care Medicine,Pulmonary and Respiratory Medicine

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