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Fatty acid amide hydrolase-dependent generation of antinociceptive drug metabolites acting on TRPV1 in the brain

PloS one, 2013-08, Vol.8 (8), p.e70690

2013

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Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

The New England journal of medicine, 2017-11, Vol.377 (21), p.2013-2023

2017

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Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

The New England journal of medicine, 2017-11, Vol.377 (21), p.2024-2035

2017

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Structural identification of a hotspot on CFTR for potentiation

Science (American Association for the Advancement of Science), 2019-06, Vol.364 (6446), p.1184-1188

2019

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A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

The New England journal of medicine, 2011-11, Vol.365 (18), p.1663-1672

2011

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Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis

Science translational medicine, 2014-07, Vol.6 (246), p.246ra96

2014

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Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Science translational medicine, 2016-06, Vol.8 (344), p.344ra84

2016

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Modulators of CFTR. Updates on clinical development and future directions

European journal of medicinal chemistry, 2021-03, Vol.213, p.113195-113195, Article 113195

2021

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Cystic fibrosis gene modifier SLC26A9 modulates airway response to CFTR-directed therapeutics

Human molecular genetics, 2016-10, Vol.25 (20), p.4590-4600

2016

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Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data

American journal of respiratory and critical care medicine, 2015-10, Vol.192 (7), p.836-842

2015

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Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis

American journal of respiratory and critical care medicine, 2014-07, Vol.190 (2), p.175-184

2014

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Identification of A Novel Small-Molecule Binding Site of the Fat Mass and Obesity Associated Protein (FTO)

Journal of medicinal chemistry, 2015-09, Vol.58 (18), p.7341-7348

2015

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Discovery of N‑(2,4-Di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, Ivacaftor), a Potent and Orally Bioavailable CFTR Potentiator

Journal of medicinal chemistry, 2014-12, Vol.57 (23), p.9776-9795

2014

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Asymmetric Synthesis and Biological Evaluation of Platensilin, Platensimycin, Platencin, and Their Analogs via a Bioinspired Skeletal Reconstruction Approach

Journal of the American Chemical Society, 2024-07, Vol.146 (28), p.18967-18978

2024

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Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

Proceedings of the National Academy of Sciences - PNAS, 2009-11, Vol.106 (44), p.18825-18830

2009

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Pharmacological Improvement of Cystic Fibrosis Transmembrane Conductance Regulator Function Rescues Airway Epithelial Homeostasis and Host Defense in Children with Cystic Fibrosis

American journal of respiratory and critical care medicine, 2024-06, Vol.209 (11), p.1338-1350

2024

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Ion Channel Modulators in Cystic Fibrosis

Chest, 2018-08, Vol.154 (2), p.383-393

2018

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Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment

Scientific reports, 2021-10, Vol.11 (1), p.19810-19810, Article 19810

2021

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Theratyping cystic fibrosis in vitro in ALI culture and organoid models generated from patient-derived nasal epithelial conditionally reprogrammed stem cells

The European respiratory journal, 2021-12, Vol.58 (6), p.2100908

2021

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Real-Life Experience with CFTR Modulators Shows Correction of LAD-IV Phenotype in Cystic Fibrosis

American journal of respiratory cell and molecular biology, 2024-10, Vol.71 (4), p.495-498

2024

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Elexacaftor/Ivacaftor/Tezacaftor: First Approval

Drugs (New York, N.Y.), 2019-12, Vol.79 (18), p.2001-2007

Hoy, Sheridan M.

2019

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Impact of CFTR Modulation on Pseudomonas aeruginosa Infection in People With Cystic Fibrosis

The Journal of infectious diseases, 2024-09, Vol.230 (3), p.e536-e547

2024

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Repression of GSK3 restores NK cell cytotoxicity in AML patients

Nature communications, 2016-04, Vol.7 (1), p.11154-11154, Article 11154

2016

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Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

Science translational medicine, 2014-07, Vol.6 (246), p.246ra97

2014

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Pharmacological approaches for targeting cystic fibrosis nonsense mutations

European journal of medicinal chemistry, 2020-08, Vol.200, p.112436-112436, Article 112436

2020

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Fatty acid amide hydrolase-dependent generation of antinociceptive drug metabolites acting on TRPV1 in the brain

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Structural identification of a hotspot on CFTR for potentiation

A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Modulators of CFTR. Updates on clinical development and future directions

Cystic fibrosis gene modifier SLC26A9 modulates airway response to CFTR-directed therapeutics

Sustained Benefit from ivacaftor demonstrated by combining clinical trial and cystic fibrosis patient registry data

Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis

Identification of A Novel Small-Molecule Binding Site of the Fat Mass and Obesity Associated Protein (FTO)

Discovery of N‑(2,4-Di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, Ivacaftor), a Potent and Orally Bioavailable CFTR Potentiator

Asymmetric Synthesis and Biological Evaluation of Platensilin, Platensimycin, Platencin, and Their Analogs via a Bioinspired Skeletal Reconstruction Approach

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

Pharmacological Improvement of Cystic Fibrosis Transmembrane Conductance Regulator Function Rescues Airway Epithelial Homeostasis and Host Defense in Children with Cystic Fibrosis

Ion Channel Modulators in Cystic Fibrosis

Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment

Theratyping cystic fibrosis in vitro in ALI culture and organoid models generated from patient-derived nasal epithelial conditionally reprogrammed stem cells

Real-Life Experience with CFTR Modulators Shows Correction of LAD-IV Phenotype in Cystic Fibrosis

Elexacaftor/Ivacaftor/Tezacaftor: First Approval

Impact of CFTR Modulation on Pseudomonas aeruginosa Infection in People With Cystic Fibrosis

Repression of GSK3 restores NK cell cytotoxicity in AML patients

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

Pharmacological approaches for targeting cystic fibrosis nonsense mutations

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