Results 1 to 10 of about 12,467 (245)

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

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   +2 more sources

Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer [PDF]

Therapeutic Advances in Medical Oncology
Background: Drug repurposing has emerged as an effective strategy to accelerate drug discovery. Using the pipeline established from a large collaborative drug repurposing project focused on high-grade serous ovarian cancer (HGSOC), we identified ...
Dongli Liu, Michelle Wong-Brown, Farhana Amy Sarker, Bayley Matthews, Jessica Morrison, Kristie-Ann Dickson, Amani Alghalayini, Jana Stojanova, King Man Wan, Jennifer Duggan, Christine Loo, Gill Stannard, Elyse Powell, Brigitte Hodder, Ellen Barlow, Deborah J. Marsh, Caroline E. Ford, Nikola A. Bowden   +17 more
doaj   +2 more sources

SLC26A9 Gene Is Associated With Lung Function Response to Ivacaftor in Patients With Cystic Fibrosis

Frontiers in Pharmacology, 2018
Ivacaftor is a drug used to treat cystic fibrosis (CF) patients carrying specific gating CFTR mutations. Interpatient variability in the lung response has been shown to be partly explained by rs7512462 in the Solute Carrier Family 26 Member 9 (SLC26A9 ...
Harriet Corvol, Harriet Corvol, Julie Mésinèle, Isman-Hassan Douksieh, Isman-Hassan Douksieh, Lisa J. Strug, Pierre-Yves Boëlle, Loïc Guillot   +7 more
doaj   +2 more sources

Effect of CFTR modulators on glucose homeostasis in children and young adults with cystic fibrosis-related diabetes: a systematic review [PDF]

Frontiers in Endocrinology
IntroductionCystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CFTR gene, leading to impaired chloride transport, thickened mucus, and multiorgan dysfunction. Among its complications, cystic fibrosis-related diabetes (CFRD)
Paola Giordano, Giuseppina Leonetti, Vanja Granberg, Rosa Maria Pia Casolino, Giuseppe Lassandro, Maurizio Delvecchio, Giovanna Linguiti   +6 more
doaj   +2 more sources

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

American Journal of Respiratory and Critical Care Medicine, 2014
Sonya L Heltshe, Scott H Donaldson, Scott D Sagel   +2 more
exaly   +2 more sources

Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR [PDF]

, 2017
Jennifer L. Taylor‐Cousar, Manu Jain, Tara Lynn Barto, Tarik Haddad, Jeffrey J. Atkinson, Simon Tian, Rui Tang, Gautham Marigowda, David Waltz, Joseph M. Pilewski   +9 more
openalex   +4 more sources

Cutaneous Adverse Effects of Elexacaftor-Tezacaftor-Ivacaftor: A Single Center Cohort Study on Acne Severity in Adults With Cystic Fibrosis. [PDF]

J Cosmet Dermatol
Smith AD, Lyons CE, Somerville L, Flowers RH, Kaplan E, Albon D.   +5 more
europepmc   +3 more sources

Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data.

PLoS ONE, 2023
ObjectivesCystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific gene mutations ...
Lisa J McGarry, Zahra Bhaiwala, Andrea Lopez, Conor Chandler, Christopher G Pelligra, Jaime L Rubin, Theodore G Liou   +6 more
doaj   +2 more sources

Urticaria multiforme-like eruption due to a novel agent elexacaftor/tezacaftor/ivacaftor in a pediatric patient with cystic fibrosis

JAAD Case Reports, 2021
Rebecca H. Goldberg, BS, Natalie H. Matthews, MD, MPhil, Alexandra C. Hristov, MD, Frank Wang, MD   +3 more
doaj   +2 more sources

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