Background: Cystic fibrosis (CF) patients have reduced intestinal absorption of sterols and, despite enhanced endogenous synthesis, low plasma cholesterol. Lumacaftor/ivacaftor CFTR protein modulator therapy is used to improve the clinical outcome of CF patients homozygous for F508del mutation (homo-deltaF508). Aim of the study is to evaluate the cholesterol metabolism and hepatobiliary injury/function in adult homo-deltaF508 patients, before and after lumacaftor/ivacaftor treatment. Baseline parameters in homo-deltaF508 patients were compared to those in CF patients compound heterozygous for F508del mutation and another severe mutation (hetero-deltaF508). Methods: Cholesterol metabolism was evaluated measuring plasma phytosterols and chole...
International audienceRationale: Lumacaftor-ivacaftor is a CFTR (cystic fibrosis transmembrane condu...
BACKGROUND: Low cholesterol is typically observed in the plasma of patients with cystic fibrosis (CF...
Objective: Disruption of the enterohepatic circulation of bile acids (BAs) is part of the gastrointe...
Background: Cystic fibrosis (CF) patients have reduced intestinal absorption of sterols and, despite...
: The last ten years have been characterized by an enormous step forward in the therapy and manageme...
Aims In clinical trials, elevated liver transaminase levels in patients taking lumacaftor/ivacaftor...
BACKGROUND: Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cyst...
In clinical trials, elevated liver transaminase levels in patients taking lumacaftor/ivacaftor thera...
Rationale: In a prior study, lumacaftor/ivacaftor treatment (<= 28 d) in patients with cystic fibros...
Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, mos...
International audienceRationale: Lumacaftor-ivacaftor is a CFTR (cystic fibrosis transmembrane condu...
BACKGROUND: Low cholesterol is typically observed in the plasma of patients with cystic fibrosis (CF...
Objective: Disruption of the enterohepatic circulation of bile acids (BAs) is part of the gastrointe...
Background: Cystic fibrosis (CF) patients have reduced intestinal absorption of sterols and, despite...
: The last ten years have been characterized by an enormous step forward in the therapy and manageme...
Aims In clinical trials, elevated liver transaminase levels in patients taking lumacaftor/ivacaftor...
BACKGROUND: Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cyst...
In clinical trials, elevated liver transaminase levels in patients taking lumacaftor/ivacaftor thera...
Rationale: In a prior study, lumacaftor/ivacaftor treatment (<= 28 d) in patients with cystic fibros...
Cystic fibrosis (CF) is a genetic disorder that causes multiorgan morbidity and premature death, mos...
International audienceRationale: Lumacaftor-ivacaftor is a CFTR (cystic fibrosis transmembrane condu...
BACKGROUND: Low cholesterol is typically observed in the plasma of patients with cystic fibrosis (CF...
Objective: Disruption of the enterohepatic circulation of bile acids (BAs) is part of the gastrointe...