Results 11 to 20 of about 2,457 (125)

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

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Frontiers in Pharmacology, 2021
Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare
Khadidja Abdallah, Kris De Boeck, Marc Dooms, Steven Simoens   +3 more
doaj   +1 more source

Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis

eLife, 2020
Previously, we showed that serum and monocytes from patients with CF exhibit an enhanced NLRP3-inflammasome signature with increased IL-18, IL-1β, caspase-1 activity and ASC speck release (Scambler et al. eLife 2019).
Heledd H Jarosz-Griffiths, Thomas Scambler, Chi H Wong, Samuel Lara-Reyna, Jonathan Holbrook, Fabio Martinon, Sinisa Savic, Paul Whitaker, Christine Etherington, Giulia Spoletini, Ian Clifton, Anil Mehta, Michael F McDermott, Daniel Peckham   +13 more
doaj   +1 more source

In Silico Screening of Novel TMPRSS2 Inhibitors for Treatment of COVID-19

Molecules, 2022
COVID-19, a pandemic caused by the virus SARS-CoV-2, has spread globally, necessitating the search for antiviral compounds. Transmembrane protease serine 2 (TMPRSS2) is a cell surface protease that plays an essential role in SARS-CoV-2 infection ...
Shuo Wang, Xuexun Fang, Ye Wang
doaj   +1 more source

Nationwide lung function monitoring from infancy in newborn-screened children with cystic fibrosis

ERJ Open Research, 2023
Background Cystic fibrosis (CF) lung disease starts in infancy and can be assessed for structural lung abnormalities using computed tomography or magnetic resonance scans, or for lung function impairment using multiple breath washout (MBW).
Rikke M. Sandvik, Marika N. Schmidt, Christian M. Voldby, Frederik F. Buchvald, Hanne V. Olesen, Jørgen Olsen, Maja V. Kragh, Sune L.M. Rubak, Tacjana Pressler, Paul D. Robinson, Per M. Gustafsson, Marianne Skov, Kim G. Nielsen   +12 more
doaj   +1 more source