VOCAL: An observational study of ivacaftor for people with cystic fibrosis and selected non-G551D-CFTR gating mutations.

Nicholas J. Simmonds,C. Kors van der Ent,Carla Colombo,Nils Kinnman,Cynthia DeSouza,Teja Thorat, Marci L. Chew,Keval Chandarana,Carlo Castellani

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society（2023）

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摘要

These results demonstrate the positive long-term effectiveness of ivacaftor on clinical outcomes and HCRU in pwCF with non-G551D-CFTR gating mutations in real-world settings.

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关键词

CFTR gating mutation,Cystic fibrosis,Healthcare resource utilization,Ivacaftor,Lung function,Real-world experience

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