Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial (Q46415131)

22 December 2017

title

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

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22 December 2017

main subject

phase II clinical trial

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8

Hannah Blau

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22 December 2017

author name string

Eitan Kerem

1

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22 December 2017

Samit Hirawat

2

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22 December 2017

Shoshana Armoni

3

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22 December 2017

Yasmin Yaakov

4

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22 December 2017

David Shoseyov

5

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22 December 2017

Michael Cohen

6

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22 December 2017

Malka Nissim-Rafinia

7

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22 December 2017

Joseph Rivlin

9

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22 December 2017

Micha Aviram

10

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22 December 2017

Gary L Elfring

11

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22 December 2017

Valerie J Northcutt

12

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22 December 2017

Langdon L Miller

13

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22 December 2017

Batsheva Kerem

14

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22 December 2017

Michael Wilschanski

15

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22 December 2017

language of work or name

English

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publication date

20 August 2008

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22 December 2017

published in

The Lancet

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22 December 2017

volume

372

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22 December 2017

issue

9640

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22 December 2017

page(s)

719-727

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22 December 2017

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

cites work

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Cystic fibrosis in Jews: frequency and mutation distribution

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

CFTR Genotype as a Predictor of Prognosis in Cystic Fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Aminoglycoside suppression of a premature stop mutation in a Cftr-/- mouse carrying a human CFTR-G542X transgene.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Gentamicin-Induced Correction of CFTR Function in Patients with Cystic Fibrosis andCFTRStop Mutations

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Nonsense-mediated mRNA decay affects nonsense transcript levels and governs response of cystic fibrosis patients to gentamicin

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

PTC124 targets genetic disorders caused by nonsense mutations

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

In vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Standardized procedure for measurement of nasal potential difference: an outcome measure in multicenter cystic fibrosis clinical trials

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Nasal potential difference measurements in patients with atypical cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Basic protocol for transepithelial nasal potential difference measurements

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Increased bioelectric potential difference across respiratory epithelia in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Reproducibility of nasal potential difference measurements in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Pharmacologic modulation of salt and water in the airway epithelium in cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Evidence of CFTR function in cystic fibrosis after systemic administration of 4-phenylbutyrate

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Effect of oral digoxin, topical ouabain and salbutamol on transepithelial nasal potential difference in patients with cystic fibrosis

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

The effects of fluticasone propionate on nasal epithelial potential difference.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

A multicenter study of the effect of solution temperature on nasal potential difference measurements

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Acidic pH hyperpolarizes nasal potential difference

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Identification and characterization of mutations in the UPF1 gene that affect nonsense suppression and the formation of the Upf protein complex but not mRNA turnover

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Quality control of eukaryotic mRNA: safeguarding cells from abnormal mRNA function

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

The efficiency of nonsense-mediated mRNA decay is an inherent character and varies among different cells

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Nasal airway ion transport is linked to the cystic fibrosis phenotype in adult patients.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

Chloride transport in nasal ciliated cells of cystic fibrosis heterozygotes.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

A novel mutation in the cystic fibrosis gene in patients with pulmonary disease but normal sweat chloride concentrations

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961168-X

7 January 2021

10.1016/S0140-6736(08)61168-X

Identifiers

DOI

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22 December 2017

PubMed ID

18722008

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22 December 2017