Vx-659-Tezacaftor-Ivacaftor In Patients With Cystic Fibrosis And One Or Two Phe508del Alleles

Erica Merman,Matthew B. Stanbrook,Anju Anand

CANADIAN JOURNAL OF RESPIRATORY CRITICAL CARE AND SLEEP MEDICINE（2020）

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摘要

Cystic fibrosis is an autosomal-recessive, multisystem disease that affects ∼80,000 patients worldwide.1,2 It is caused by mutations in the CFTR gene, an epithelial chloride ion channel with varyin...

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cystic fibrosis,phe508del alleles,vx-659–tezacaftor–ivacaftor,vx-659–tezacaftor–ivacaftor

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