Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial (Q34132007)

31 July 2017

title

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial (English)

1 reference

31 July 2017

main subject

phase III clinical trial

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Isabelle Fajac

10

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Pamela Zeitlin

object named as

Pamela L Zeitlin

18

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Stuart Elborn

object named as

J Stuart Elborn

7

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Richard Moss

1 reference

ORCID Public Data File 2021

Isabelle Sermet-Gaudelus

5

object named as

Isabelle Sermet-Gaudelus

1 reference

31 July 2017

Eitan Kerem

1

1 reference

31 July 2017

Michael W Konstan

2

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31 July 2017

Kris De Boeck

3

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Frank J Accurso

4

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Michael Wilschanski

6

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Paola Melotti

8

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31 July 2017

Inez Bronsveld

9

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Anne Malfroot

11

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Daniel B Rosenbluth

12

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Patricia A Walker

13

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Susanna A McColley

14

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Christiane Knoop

15

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31 July 2017

Serena Quattrucci

16

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31 July 2017

Ernst Rietschel

17

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Jay Barth

19

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Gary L Elfring

20

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Ellen M Welch

21

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Arthur Branstrom

22

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Robert J Spiegel

23

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Stuart W Peltz

24

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Temitayo Ajayi

25

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31 July 2017

Steven M Rowe

26

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31 July 2017

Cystic Fibrosis Ataluren Study Group

27

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31 July 2017

publication date

15 May 2014

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The Lancet. Respiratory medicine

31 July 2017

published in

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31 July 2017

volume

2

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31 July 2017

issue

7

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31 July 2017

page(s)

539-547

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A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect.

31 July 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Ataluren as an agent for therapeutic nonsense suppression

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Rescue of nonsense mutations by amlexanox in human cells

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

PTC124 targets genetic disorders caused by nonsense mutations

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Standardisation of spirometry

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

General considerations for lung function testing.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Genotype-phenotype correlation for pulmonary function in cystic fibrosis.

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Spirometric reference values from a sample of the general U.S. population

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Genotype-phenotype correlations in cystic fibrosis

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Firefly luciferase as a tool in molecular and cell biology

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Association of a nonsense mutation (W1282X), the most common mutation in the Ashkenazi Jewish cystic fibrosis patients in Israel, with presentation of severe disease

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

5 July 2018

Tobramycin: an overview

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

A Method for Testing for Synergy with Any Number of Agents

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis

5 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Antibiotic treatment of initial colonization with Pseudomonas aeruginosa postpones chronic infection and prevents deterioration of pulmonary function in cystic fibrosis

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Pulmonary function between 6 and 18 years of age.

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

CFTR Genotype as a Predictor of Prognosis in Cystic Fibrosis

28 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4154311

Correlation between genotype and phenotype in patients with cystic fibrosis

28 October 2018

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2814%2970100-6

7 January 2021

Increased bioelectric potential difference across respiratory epithelia in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2814%2970100-6

7 January 2021

Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2814%2970100-6

7 January 2021

Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2814%2970100-6

7 January 2021

A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2814%2970100-6

7 January 2021

A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function

1 reference

https://api.crossref.org/works/10.1016%2FS2213-2600%2814%2970100-6

7 January 2021

Measurements of nasal transepithelial electric potential differences in normal human subjects in vivo

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24836205

12 December 2020

inferred from PubMed ID database lookup

Identifiers

DOI

10.1016/S2213-2600(14)70100-6

1 reference

31 July 2017

1 reference

31 July 2017

1 reference