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Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis [PDF]

open access: yesCPT: Pharmacometrics & Systems Pharmacology
Ivacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF ...
Ngoc Hoa Truong   +16 more
doaj   +3 more sources

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

open access: yesFrontiers 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   +12 more
doaj   +2 more sources

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

open access: yesBMJ 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   +3 more
doaj   +2 more sources

Adherence to Ivacaftor is suboptimal [PDF]

open access: yesJournal of Cystic Fibrosis, 2015
Ivacaftor, the first cystic fibrosis (CF) transmembrane conductance regulator (CFTR) potentiator provides a remarkable example of personalised medicine that has the capacity to transform lives and patient care. For those with a CFTR-G551D mutation, two phase three trials (adult and paediatric age 6+ years) and a follow-up open label trial have ...
Abbott, Janice, Bilton, Diana
openaire   +4 more sources

The magnitude of ivacaftor effects on fluid secretion via R117H-CFTR channels: Human in vivo measurements.

open access: yesPLoS ONE, 2017
We optically measured effects of orally available ivacaftor (Kalydeco®) on sweat rates of identified glands in 3 R117H subjects, each having a unique set of additional mutations, and compared them with 5 healthy control subjects tested contemporaneously.
Jessica E Char   +5 more
doaj   +2 more sources

Pharmacokinetic and Drug–Drug Interaction Profiles of the Combination of Tezacaftor/Ivacaftor

open access: yesClinical and Translational Science, 2019
Drug–drug interaction (DDI) studies are described for tezacaftor/ivacaftor, a new cystic fibrosis transmembrane conductance regulator modulator therapy for the treatment of cystic fibrosis.
Varun Garg   +11 more
doaj   +2 more sources

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

open access: yesTherapeutic 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   +17 more
doaj   +2 more sources

The potentially beneficial central nervous system activity profile of ivacaftor and its metabolites [PDF]

open access: yesERJ Open Research, 2018
Ivacaftor–lumacaftor and ivacaftor are two new breakthrough cystic fibrosis transmembrane conductance modulators. The interactions of ivacaftor and its two metabolites hydroxymethylivacaftor (iva-M1) and ivacaftorcarboxylate (iva-M6) with ...
Elena K. Schneider   +10 more
doaj   +2 more sources

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

open access: yesFrontiers 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   +7 more
doaj   +2 more sources

Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience [PDF]

open access: yesPulmonary Therapy, 2020
Plain Language Summary We performed a study to better understand the long-term impact of treatment with a drug called ivacaftor for patients with cystic fibrosis (CF).
Mark Higgins   +4 more
doaj   +2 more sources

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