Challenges in the use of highly effective modulator treatment for cystic fibrosis
Author:
Funder
Cystic Fibrosis Foundation
National Institutes of Health
Publisher
Elsevier BV
Subject
Pulmonary and Respiratory Medicine,Pediatrics, Perinatology, and Child Health
Reference65 articles.
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2. Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele;Middleton;N Engl J Med,2019
3. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial;Heijerman;Lancet,2019
4. Food and drug administration (FDA). TRIKAFTA drug insert. 2020 Jan 12, 2021]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/212273s002lbl.pdf.
5. Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease;Shteinberg;Eur Respir Rev,2020
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