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. (Q37102828)

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scientific article published on 08 August 2011

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English	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.	scientific article published on 08 August 2011

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9 February 2020

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 (English)

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9 February 2020

cystic fibrosis

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Michael P Boyle

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Ronald C. Rubenstein

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9 February 2020

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J P Clancy

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Steven M Rowe

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Frank J Accurso

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Moira L Aitken

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Raouf S Amin

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Melissa A Ashlock

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Manfred Ballmann

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Inez Bronsveld

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Preston W Campbell

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Kris De Boeck

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Scott H Donaldson

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Henry L Dorkin

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Jordan M Dunitz

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Peter R Durie

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Manu Jain

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Anissa Leonard

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Karen S McCoy

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Richard B Moss

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Joseph M Pilewski

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Daniel B Rosenbluth

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Michael S Schechter

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Martyn Botfield

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Claudia L Ordoñez

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George T Spencer-Green

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Laurent Vernillet

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Steve Wisseh

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Karl Yen

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Michael W Konstan

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9 February 2020

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8 August 2011

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12-18

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9 February 2020

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3746507

14 September 2017

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3746507

14 September 2017

Pharmaceuticals targeting nonsense mutations in genetic diseases: progress in development

1 reference

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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

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Detection of cystic fibrosis transmembrane conductance regulator activity in early-phase clinical trials

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Cystic fibrosis: a review of epidemiology and pathobiology

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In vitro prediction of stop-codon suppression by intravenous gentamicin in patients with cystic fibrosis: a pilot study

1 reference

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Small-molecule correctors of defective DeltaF508-CFTR cellular processing identified by high-throughput screening.

1 reference

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14 September 2017

Cystic fibrosis: a worldwide analysis of CFTR mutations--correlation with incidence data and application to screening

1 reference

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14 September 2017

Chloride conductance and genetic background modulate the cystic fibrosis phenotype of Delta F508 homozygous twins and siblings

1 reference

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ATPase activity of the cystic fibrosis transmembrane conductance regulator

1 reference

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Bicarbonate conductance and pH regulatory capability of cystic fibrosis transmembrane conductance regulator

1 reference

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Changes in the normal maximal expiratory flow-volume curve with growth and aging

1 reference

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Relative ion permeability of normal and cystic fibrosis nasal epithelium

1 reference

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Chloride impermeability in cystic fibrosis

1 reference

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Identification of the cystic fibrosis gene: genetic analysis

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Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA

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Identification and regulation of the cystic fibrosis transmembrane conductance regulator-generated chloride channel

1 reference

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A principal role for the proteasome in endoplasmic reticulum-associated degradation of misfolded intracellular cystic fibrosis transmembrane conductance regulator

1 reference

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

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27 June 2018

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1 reference

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27 June 2018

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

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3 September 2018

Development and validation of The Cystic Fibrosis Questionnaire in the United States: a health-related quality-of-life measure for cystic fibrosis.

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Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

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1 reference

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12 December 2020

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10.1136/THORAXJNL-2011-200393

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9 February 2020

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9 February 2020

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