Results 11 to 20 of about 2,581 (157)

Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis

Hepatology Communications, 2023
The cystic fibrosis (CF) transmembrane conductance regulator corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition.
Steven Levitte, Yonathan Fuchs, Russell Wise, Zachary M. Sellers   +3 more
doaj   +1 more source

Insights Into Patient Variability During Ivacaftor-Lumacaftor Therapy in Cystic Fibrosis

Frontiers in Pharmacology, 2021
Background: The advent of cystic fibrosis transmembrane conductance regulator protein (CFTR) modulators like ivacaftor have revolutionised the treatment of cystic fibrosis (CF).
Patrick O. Hanafin, Isabelle Sermet-Gaudelus, Matthias Griese, Matthias Kappler, Helmut Ellemunter, Carsten Schwarz, Carsten Schwarz, John Wilson, John Wilson, Marsha Tan, Tony Velkov, Gauri G. Rao, Elena K. Schneider-Futschik   +12 more
doaj   +1 more source

Effects of lumacaftor—ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2–11 years

Frontiers in Pharmacology, 2023
Rationale: Lumacaftor/ivacaftor was approved for the treatment of patients with cystic fibrosis who are homozygous for F508del aged 2 years and older following positive results from phase three trials. However, the improvement in CFTR function associated
Julian Berges, Julian Berges, Simon Y. Graeber, Simon Y. Graeber, Simon Y. Graeber, Susanne Hämmerling, Susanne Hämmerling, Yin Yu, Yin Yu, Arne Krümpelmann, Arne Krümpelmann, Mirjam Stahl, Mirjam Stahl, Mirjam Stahl, Stephanie Hirtz, Stephanie Hirtz, Heike Scheuermann, Heike Scheuermann, Marcus A. Mall, Marcus A. Mall, Marcus A. Mall, Olaf Sommerburg, Olaf Sommerburg   +22 more
doaj   +1 more source

Utility of Fecal Elastase-1 in Estimating Exocrine Pancreatic Function in Cystic Fibrosis: A Scoping Review. [PDF]

Pediatr Pulmonol
ABSTRACT Background In people with cystic fibrosis (pwCF), identification of exocrine pancreatic insufficiency (EPI) is essential to prevent steatorrhea and, if not managed actively, can lead to catastrophic consequences. Fecal elastase‐1 (FE‐1) is a widely used test to screen for EPI in cystic fibrosis (CF).
Sankararaman S, Hendrix S, Neudecker M, Borowitz D.   +3 more
europepmc   +2 more sources

The use of targeted therapy lumacaftor/ivacaftor in patients with cystic fibrosis

Медицинский совет, 2022
Accepted, basic therapy of cystic fibrosis (CF), until recently, was symptomatic and aimed at slowing down pathological processes, mainly from the respiratory system and gastrointestinal tract, caused by a defect in the CFTR gene.
A. G. Chermensky, T. E. Gembitskaya, A. V. Orlov, V. R. Makhmutova   +3 more
doaj   +1 more source

Clinical Efficacy and Safety of Ivacaftor/Lumacaftor Combination in Patients with Cystic Fibrosis: International Studies Review

Вопросы современной педиатрии, 2021
Cystic fibrosis is an autosomal recessive disease caused by structure abnormalities in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Nataliya Yu. Kashirskaya, Nika V. Petrova, Rena A. Zinchenko   +2 more
doaj   +1 more source

Gene Therapy: A Possible Alternative to CFTR Modulators?

Frontiers in Pharmacology, 2021
Cystic fibrosis (CF) is a rare genetic disease that affects several organs, but lung disease is the major cause of morbidity and mortality. The gene responsible for CF, the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, has been ...
J. Mercier, M. Ruffin, H. Corvol, H. Corvol, L. Guillot   +4 more
doaj   +1 more source

Long-term effects of lumacaftor/ivacaftor on paranasal sinus abnormalities in children with cystic fibrosis detected with magnetic resonance imaging

Frontiers in Pharmacology, 2023
Introduction: Chronic rhinosinusitis (CRS) usually presents with nasal congestion, rhinorrhea and anosmia impacts quality of life in cystic fibrosis (CF).
Lena Wucherpfennig, Lena Wucherpfennig, Lena Wucherpfennig, Felix Wuennemann, Felix Wuennemann, Felix Wuennemann, Felix Wuennemann, Monika Eichinger, Monika Eichinger, Monika Eichinger, Angelika Seitz, Ingo Baumann, Mirjam Stahl, Mirjam Stahl, Mirjam Stahl, Simon Y. Graeber, Simon Y. Graeber, Simon Y. Graeber, Shengkai Zhao, Shengkai Zhao, Jaehi Chung, Jaehi Chung, Jens-Peter Schenk, Abdulsattar Alrajab, Hans-Ulrich Kauczor, Hans-Ulrich Kauczor, Hans-Ulrich Kauczor, Marcus A. Mall, Marcus A. Mall, Marcus A. Mall, Olaf Sommerburg, Olaf Sommerburg, Mark O. Wielpütz, Mark O. Wielpütz, Mark O. Wielpütz   +34 more
doaj   +1 more source

Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis

EBioMedicine, 2015
Background: The best investigational drug to treat cystic fibrosis (CF) patients with the most common CF-causing mutation (F508del) is VX-809 (lumacaftor) which recently succeeded in Phase III clinical trial in combination with ivacaftor.
Nikhil T. Awatade, Inna Uliyakina, Carlos M. Farinha, Luka A. Clarke, Karina Mendes, Amparo Solé, Juan Pastor, Maria Margarida Ramos, Margarida D. Amaral   +8 more
doaj   +1 more source

Physiologically Based Pharmacokinetic Modeling of CFTR Modulation in People with Cystic Fibrosis Transitioning from Mono or Dual Regimens to Triple-Combination Elexacaftor/Tezacaftor/Ivacaftor

Pulmonary Therapy, 2020
Introduction The triple-combination (TC) cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen elexacaftor, tezacaftor, and ivacaftor was shown to be safe and efficacious in phase 3 trials of people with cystic fibrosis (pwCF) ≥ 12 
Alice Tsai, Shu-Pei Wu, Eric Haseltine, Sanjeev Kumar, Samuel M. Moskowitz, Paul Panorchan, Kushal Shah   +6 more
doaj   +1 more source