Real-world effectiveness of ivacaftor in children with cystic fibrosis and the G551D mutation

• Main Authors: David Gomez-Pastrana (Author), Chinedu Nwokoro (Author), Mike McLean (Author), Sarah Brown (Author), Nanna Christiansen (Author), Caroline S. Pao (Author)
• Format: Book
• Published: Elsevier, 2019-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Introduction: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has been shown to improve the nutritional status and lung function of cystic fibrosis patients with the G551D mutation in clinical trials. The objective of this study was to describe the real-wor...