Effects of Lumacaftor/Ivacaftor on Cystic Fibrosis Disease Progression in Children 2 through 5 Years of Age Homozygous for F508del-CFTR: A Phase 2 Placebo-controlled Clinical Trial

Author:

Stahl Mirjam123ORCID,Roehmel Jobst1,Eichinger Monika456,Doellinger Felix7,Naehrlich Lutz89,Kopp Matthias V.1011,Dittrich Anna-Maria1213ORCID,Lee Christopher14,Sommerburg Olaf415,Tian Simon14,Xu Tu14,Wu Pan14,Joshi Aniket14,Ray Partha14,Duncan Margaret E.14,Wielpütz Mark O.456ORCID,Mall Marcus A.123ORCID

Affiliation:

1. Department of Pediatric Respiratory Medicine, Immunology, and Critical Care Medicine and

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

3. Berlin Institute of Health at Charité – Universitätsmedizin Berlin, Berlin, Germany;

4. Translational Lung Research Center Heidelberg, German Center for Lung Research, Heidelberg, Germany;

5. Department of Diagnostic and Interventional Radiology,

6. Department of Diagnostic and Interventional Radiology with Nuclear Medicine, Thoraxklinik, and

7. Department of Radiology, Charité – Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany;

8. Department of Pediatrics, Justus Liebig University Giessen, Giessen, Germany;

9. Universities of Giessen and Marburg Lung Center, German Center for Lung Research, Giessen, Germany;

10. Pediatric Respiratory Medicine, Department of Pediatrics, Inselspital, University of Bern, Bern, Switzerland;

11. Airway Research Center North, German Center for Lung Research, Grosshansdorf, Germany;

12. Department for Pediatric Pulmonology, Allergology, and Neonatology and

13. BREATH, German Center for Lung Research, Hannover Medical School, Hannover, Germany; and

14. Vertex Pharmaceuticals Incorporated, Boston, Massachusetts

15. Division of Pediatric Pulmonology and Allergy and Cystic Fibrosis Center, Department of Pediatrics, Heidelberg University Hospital, Heidelberg, Germany;

Funder

Vertex Pharmaceuticals

Publisher

American Thoracic Society

Subject

Pulmonary and Respiratory Medicine

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