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Elexacaftor–Tezacaftor–Ivacaftor Therapy for Cystic Fibrosis Patients with the F508del/Unknown Genotype

2021-05-31

Abstract excerpt

The new CFTR modulator combination, elexacaftor/tezacaftor/ivacaftor (Trikafta) was approved by the FDA in October 2019 for treatment of Cystic Fibrosis in patients 12 years of age or older who have at least one F508del mutation in one allele and a minimal-function or another F508del mutation in the other allele. However, there is a group of patients, in addition to those with rare mutations, in which despite the...

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Literature Corpus work
46d9d966-820e-5be8-8cde-0ba193341ef0
DOI
10.20944/preprints202105.0728.v1
Open publication

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Elexacaftor–Tezacaftor–Ivacaftor Therapy for Cystic Fibrosis Patients with the F508del/Unknown GenotypeDOI 10.20944/preprints202105.0728.v1
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