Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.

The New England journal of medicine - 23 Nov 2017

Rowe Steven M, Daines Cori, Ringshausen Felix C, Kerem Eitan, Wilson John, Tullis Elizabeth, Nair Nitin, Simard Christopher, Han Linda, Ingenito Edward P, McKee Charlotte, Lekstrom-Himes Julie, Davies Jane C

Abstract excerpt

BACKGROUND: Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show residual function and respond to the CFTR potentiator ivacaftor in vitro, whereas ivacaftor alone does not restore activity to Phe508del mutant CFTR. METHODS: We conducted a randomized, double-blind, placebo-controlled, phase 3, crossover...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Aminophenols
Share to Benzodioxoles

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Cross-Over Studies

Share to Double-Blind Method

Share to Drug Combinations

Share to Forced Expiratory Volume

Share to Quality of Life

Share to Quinolones

Source

Open in PubMed Open full text

Identifiers

PMID: 29099333
DOI: 10.1056/NEJMoa1709847
PMCID: PMC6472479

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis.PMID 29099333Read ↗

Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study.Semantic proximity Read ↗

Tezacaftor for the treatment of cystic fibrosis.Semantic proximity Read ↗

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del AllelesSemantic proximity Read ↗

Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation.Semantic proximity Read ↗

Ivacaftor in cystic fibrosis with residual function: Lung function results from an N-of-1 study.Semantic proximity Read ↗

Elexacaftor-tezacaftor-ivacaftor: The new paradigm to treat people with cystic fibrosis with at least one p.Phe508del mutation.Semantic proximity Read ↗

A phase 3, double-blind, parallel-group study to evaluate the efficacy and safety of tezacaftor in combination with ivacaftor in participants 6 through 11 years of age with cystic fibrosis homozygous for F508del or heterozygous for the F508del-CFTR mutation and a residual function mutation.Semantic proximity Read ↗

Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST).Semantic proximity Read ↗

Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two<i>F508del</i>allelesSemantic proximity Read ↗

Precision Medicine Based on CFTR Genotype for People with Cystic FibrosisSemantic proximity Read ↗

Triple Therapy for Cystic Fibrosis Phe508del-Gating and -Residual Function Genotypes.Semantic proximity Read ↗

Development of elexacaftor - tezacaftor - ivacaftor: Highly effective CFTR modulation for the majority of people with Cystic Fibrosis.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.