Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis (Q47445666)

http://europepmc.org/abstract/PMC/6472479

30 June 2019

title

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis (English)

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http://europepmc.org/abstract/PMC/6472479

30 June 2019

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3

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7 June 2020

author name string

Steven M Rowe

1

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7 June 2020

Cori Daines

2

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7 June 2020

Eitan Kerem

4

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7 June 2020

John Wilson

5

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7 June 2020

Elizabeth Tullis

6

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7 June 2020

Nitin Nair

7

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7 June 2020

Christopher Simard

8

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7 June 2020

Linda Han

9

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7 June 2020

Edward P Ingenito

10

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7 June 2020

Charlotte McKee

11

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7 June 2020

Julie Lekstrom-Himes

12

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7 June 2020

Jane C Davies

13

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7 June 2020

language of work or name

English

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publication date

3 November 2017

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http://europepmc.org/abstract/PMC/6472479

30 June 2019

published in

The New England Journal of Medicine

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7 June 2020

volume

377

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7 June 2020

issue

21

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7 June 2020

page(s)

2024-2035

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Effect of genotype on phenotype and mortality in cystic fibrosis: a retrospective cohort study

7 June 2020

cites work

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12 December 2020

Late diagnosis defines a unique population of long-term survivors of cystic fibrosis

1 reference

12 December 2020

Mutations in the cystic fibrosis transmembrane regulator gene and in vivo transepithelial potentials

1 reference

12 December 2020

Patient-reported outcomes in cystic fibrosis

1 reference

12 December 2020

Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice

1 reference

12 December 2020

1 reference

12 December 2020

Determination of the minimal clinically important difference scores for the Cystic Fibrosis Questionnaire-Revised respiratory symptom scale in two populations of patients with cystic fibrosis and chronic Pseudomonas aeruginosa airway infection

1 reference

12 December 2020

Clinical phenotype of cystic fibrosis patients with the G551D mutation

1 reference

12 December 2020

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation

1 reference

12 December 2020

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.

1 reference

12 December 2020

A CFTR potentiator in patients with cystic fibrosis and the G551D mutation

1 reference

12 December 2020

Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation

1 reference

12 December 2020

Personalized medicine in cystic fibrosis: dawning of a new era

1 reference

12 December 2020

Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function

1 reference

12 December 2020

Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial

1 reference

12 December 2020

Acquired defects in CFTR-dependent β-adrenergic sweat secretion in chronic obstructive pulmonary disease

1 reference

12 December 2020

Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.

1 reference

12 December 2020

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial

1 reference

12 December 2020

Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study.

1 reference

12 December 2020

On the mechanism of gating defects caused by the R117H mutation in cystic fibrosis transmembrane conductance regulator

1 reference

12 December 2020

1 reference

12 December 2020

Lumacaftor/Ivacaftor in Patients Aged 6-11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR.

1 reference

12 December 2020

Lumacaftor/Ivacaftor Treatment of Patients with Cystic Fibrosis Heterozygous for F508del-CFTR.

1 reference

12 December 2020

Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.

1 reference

12 December 2020

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial

1 reference

12 December 2020

Tezacaftor/Ivacaftor in Subjects with Cystic Fibrosis and F508del/F508del-CFTR or F508del/G551D-CFTR.

1 reference

12 December 2020

Identifiers

DOI

10.1056/NEJMOA1709847

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29099333%20AND%20SRC:MED&resulttype=core&format=json

7 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29099333%20AND%20SRC:MED&resulttype=core&format=json

7 June 2020

PubMed publication ID

29099333

1 reference