Add to ORKGABSTRACT: In patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR) biomarkers, such as sweat chloride concentration and/or nasal potential difference, are used as end-points of efficacy in phase-III clinical trials with the disease modifying drugs ivacaftor (VX-770), VX809 and ataluren. The aim of this project was to review the literature on reliability, validity and responsiveness of nasal potential difference, sweat chloride and intestinal current measurement in patients with cystic fibrosis. Data on clinimetric properties were collected for each biomarker and reviewed by an international team of experts. Data on reliability, validity and responsiveness were tabulated. In addition, narrative answers to ...
AbstractBackgroundPreclinical data suggest that miglustat could restore the function of the cystic f...
Background: Cystic fibrosis (CF) is a hereditary disorder caused by genetic mutations, which affect ...
Abstract Background Cystic fibrosis (CF) is caused by mutations in the gene encoding for the CF tran...
In patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR) biomark...
textabstractIn patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (C...
International audienceWith the advent of CFTR modulators, surrogate outcome parameters that accurate...
AbstractBackgroundWe examined data from a Phase 2 trial {NCT00457821} of ivacaftor, a CFTR potentiat...
Cystic fibrosis, a multi-organ genetic disease, is characterized by abnormal function of the cystic ...
Cystic Fibrosis (CF) is a hereditary, systemic disease caused by mutations in the Cystic Fibrosis Tr...
BACKGROUND: In patients with symptoms suggestive of cystic fibrosis (CF) and intermediate sweat chlo...
The diagnosis of cystic fibrosis (CF) is not always certain, despite extensive clinical evaluation, ...
Background: In patients with symptoms suggestive of cystic fibrosis (CF) and intermediate sweat chlo...
Cystic Fibrosis (CF) is the most common severe genetic disease among the Caucasian population. It is...
The gold standard for diagnosing cystic fibrosis (CF) is a sweat chloride value above 60 mEq/L. Howe...
AbstractIvacaftor, a CFTR potentiator that enhances chloride transport by acting directly on CFTR to...
AbstractBackgroundPreclinical data suggest that miglustat could restore the function of the cystic f...
Background: Cystic fibrosis (CF) is a hereditary disorder caused by genetic mutations, which affect ...
Abstract Background Cystic fibrosis (CF) is caused by mutations in the gene encoding for the CF tran...
In patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR) biomark...
textabstractIn patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (C...
International audienceWith the advent of CFTR modulators, surrogate outcome parameters that accurate...
AbstractBackgroundWe examined data from a Phase 2 trial {NCT00457821} of ivacaftor, a CFTR potentiat...
Cystic fibrosis, a multi-organ genetic disease, is characterized by abnormal function of the cystic ...
Cystic Fibrosis (CF) is a hereditary, systemic disease caused by mutations in the Cystic Fibrosis Tr...
BACKGROUND: In patients with symptoms suggestive of cystic fibrosis (CF) and intermediate sweat chlo...
The diagnosis of cystic fibrosis (CF) is not always certain, despite extensive clinical evaluation, ...
Background: In patients with symptoms suggestive of cystic fibrosis (CF) and intermediate sweat chlo...
Cystic Fibrosis (CF) is the most common severe genetic disease among the Caucasian population. It is...
The gold standard for diagnosing cystic fibrosis (CF) is a sweat chloride value above 60 mEq/L. Howe...
AbstractIvacaftor, a CFTR potentiator that enhances chloride transport by acting directly on CFTR to...
AbstractBackgroundPreclinical data suggest that miglustat could restore the function of the cystic f...
Background: Cystic fibrosis (CF) is a hereditary disorder caused by genetic mutations, which affect ...
Abstract Background Cystic fibrosis (CF) is caused by mutations in the gene encoding for the CF tran...