Results 31 to 40 of about 9,288 (216)

Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease

European Respiratory Review, 2020
Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation ...
Michal Shteinberg, Jennifer L. Taylor-Cousar   +1 more
doaj   +1 more source

Regulatory T cell enhancement in adults with cystic fibrosis receiving Elexacaftor/Tezacaftor/Ivacaftor therapy

Frontiers in Immunology, 2023
IntroductionCystic fibrosis (CF), especially CF lung disease, is characterized by chronic infection, immune dysfunction including impairment of regulatory T cells (Tregs) and an exaggerated inflammatory response.
Dirk Westhölter, Jonas Raspe, Hendrik Uebner, Johannes Pipping, Mona Schmitz, Svenja Straßburg, Sivagurunathan Sutharsan, Matthias Welsner, Christian Taube, Sebastian Reuter   +9 more
doaj   +1 more source

Effect of elexacaftor/tezacaftor/ivacaftor on airway and systemic inflammation in cystic fibrosis

Thorax, 2023
Treatment with elexacaftor/tezacaftor/ivacaftor (ETI) has been shown to improve lung function in people with cystic fibrosis (PWCF). However, its biological effects remain incompletely understood.
M. Casey, C. Gabillard-Lefort, O. Mcelvaney, O. McElvaney, T. Carroll, R. Heeney, C. Gunaratnam, E. Reeves, M. Murphy, N. G. Mcelvaney   +9 more
semanticscholar   +1 more source

Inhibitory effects of calcium channel blockers nisoldipine and nimodipine on ivacaftor metabolism and their underlying mechanism

Frontiers in Pharmacology
Ivacaftor is the first potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein approved for use alone in the treatment of cystic fibrosis (CF).
Hailun Xia, Xinhao Xu, Jie Chen, Hualu Wu, Yuxin Shen, Xiaohai Chen, Ren-ai Xu, Wenzhi Wu   +7 more
doaj   +1 more source

Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20–121-102 and VX20–121-103): results from two randomised, active-controlled, phase 3 trials

The Lancet Respiratory Medicine
Summary Background The goal of cystic fibrosis transmembrane conductance regulator (CFTR) modulators is to reach normal CFTR function in people with cystic fibrosis.
C. Keating, L. Yonker, F. Vermeulen, Dario Prais, Rachel W. Linnemann, A. Trimble, T. Kotsimbos, J. Mermis, A. T. Braun, M. O'Carroll, S. Sutharsan, Bonnie W Ramsey, M. Mall, J. Taylor-Cousar, E. McKone, Elizabeth Tullis, T. Floreth, P. Michelson, P. Sosnay, N. Nair, R. Zahigian, H. Martin, N. Ahluwalia, A. Lam, A. Horsley   +24 more
semanticscholar   +1 more source

Real-world impact of ivacaftor in people with cystic fibrosis and select ivacaftor-responsive mutations

BMJ Open Respiratory Research
Background Ivacaftor approval was extended to people with cystic fibrosis (CF) with ≥1 of 28 additional ivacaftor-responsive mutations in the USA in 2017 based on preclinical in vitro data.
Mark Higgins, Teja Thorat, Craig McKinnon, Alexander Craft   +3 more
doaj   +1 more source

Determining the Minimal Clinically Important Difference of the 40‐Item Smell Identification Test in People With Cystic Fibrosis

International Forum of Allergy &Rhinology, EarlyView.
ABSTRACT Background Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are highly prevalent among people with cystic fibrosis (PwCF) and negatively impact quality of life. The 40‐item Smell Identification Test (SIT) is widely used to assess psychophysical olfaction, but a CF‐specific minimal clinically important difference (MCID) has not been ...
Eugene Oh, Jessa E. Miller, Michelle J. Lee, Anna Zemke, David Baraghoshi, Matthew J. Strand, Jeremiah A. Alt, Todd E. Bodner, Michael Chang, Naweed I. Chowdhury, Patricia H. Eshaghian, Anne E. Getz, Jennifer L. Goralski, David A. Gudis, Peter H. Hwang, Ashoke Khanwalkar, Adam J. Kimple, Jivianne T. Lee, Douglas A. Li, Jess C. Mace, Jayakar V. Nayak, Jonathan B. Overdevest, Zara Patel, Daniella K. Safatian, Rodney J. Schlosser, Brent Senior, Amanda L. Stapleton, Timothy L. Smith, Zachary M. Soler, Jeffrey D. Suh, Grant A. Turner, Marilene B. Wang, Milene T. Saavedra, Jennifer L. Taylor‐Cousar, Daniel M. Beswick   +34 more
wiley   +1 more source

Optimization of pharmaceutical research and development by early‐phase assessment of investigational medicinal products

British Journal of Clinical Pharmacology, EarlyView.
Small and mid‐sized pharmaceutical innovators often have limited in‐house health economics and market access expertise, and may struggle to align development strategies of investigational medicinal products with health system needs and payer expectations.
Zoltán Kaló, Attila Imre, Gábor Varga, Sándor Kovács, György Tímár, Balázs Nagy   +5 more
wiley   +1 more source

Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis

Pharmaceuticals, 2023
Cystic fibrosis (CF) is a potentially fatal monogenic disease that causes a progressive multisystemic pathology. Over the last decade, the introduction of CF transmembrane conductance regulator (CFTR) modulator drugs into clinical practice has profoundly
M. Bacalhau, M. Camargo, Grace A V Magalhães-Ghiotto, Sybelle Drumond, C. H. M. Castelletti, M. Lopes-Pacheco   +5 more
semanticscholar   +1 more source

Pregnancy‐related effect on elexacaftor, tezacaftor and ivacaftor pharmacokinetics in women with cystic fibrosis

British Journal of Clinical Pharmacology, EarlyView.
Aim The number of pregnancies among women with cystic fibrosis (wwCF) has steadily increased over the past decade. However, the pharmacokinetics (PK) of elexacaftor–tezacaftor–ivacaftor (ETI) during gestation remains uncharacterized, despite its widespread use in this population.
Paulette Magnas, Naim Bouazza, Frantz Foissac, Saïk Urien, Léo Froelicher‐Bournaud, Gabrielle Lui, Victoria Guillemart, Nicolas Carlier, Jennifer Da Silva, Johanna Fesenbeckh, Reem Kanaan, Isabelle Honoré, Clémence Martin, Jean‐Marc Treluyer, Vassilis Tsatsaris, Pierre‐Régis Burgel, Sihem Benaboud   +16 more
wiley   +1 more source