Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial

Author:

Sagel Scott D.1ORCID,Khan Umer2,Heltshe Sonya L.23,Clancy John P.45,Borowitz Drucy6,Gelfond Daniel7,Donaldson Scott H.8,Moran Antoinette9,Ratjen Felix10,VanDalfsen Jill M.2,Rowe Steven M.11

Affiliation:

1. Department of Pediatrics, Children’s Hospital Colorado and University of Colorado Anschutz Medical Campus, Aurora, Colorado

2. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children’s Research Institute, Seattle, Washington

3. Department of Pediatrics, School of Medicine, University of Washington, Seattle, Washington

4. Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio

5. Cystic Fibrosis Foundation, Bethesda, Maryland

6. Department of Pediatrics, Jacobs School of Medicine and

7. Western New York Pediatric Gastroenterology, University at Buffalo, Buffalo, New York

8. Department of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina

9. Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota

10. Department of Pediatrics, Hospital for Sick Children, Toronto, Ontario, Canada; and

11. Department of Medicine and the Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham, Birmingham, Alabama

Publisher

American Thoracic Society

Subject

Pulmonary and Respiratory Medicine

Reference35 articles.

1. Cystic fibrosis

2. Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA

3. Identification of the Cystic Fibrosis Gene: Genetic Analysis

4. Cystic Fibrosis

5. Cystic Fibrosis Foundation. Patient registry 2018 annual data report. Bethesda, MD: Cystic Fibrosis Foundation; 2018.

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