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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes

The New England journal of medicine, 2021-08, Vol.385 (9), p.815-825

2021

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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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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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Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study

The lancet respiratory medicine, 2019-04, Vol.7 (4), p.325-335

2019

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Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study

The lancet respiratory medicine, 2016-02, Vol.4 (2), p.107-115

2016

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An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2–5 years (KLIMB)

Journal of cystic fibrosis, 2019-11, Vol.18 (6), p.838-843

2019

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From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis

International journal of molecular sciences, 2020-08, Vol.21 (16), p.5882

2020

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Pharmacokinetic Enhancement of Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: A Cost Reduction Strategy to Address Global Disparities in Access

Clinical pharmacology and therapeutics, 2024-06, Vol.115 (6), p.1204-1207

2024

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In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N‐of‐1 studies

Pediatric pulmonology, 2017-04, Vol.52 (4), p.472-479

2017

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Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis

Journal of cystic fibrosis, 2024-05, Vol.23 (3), p.429-435

2024

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1-BENZYLSPIRO[PIPERIDINE-4,1′-PYRIDO[3,4-b]indole] ‘co-potentiators’ for minimal function CFTR mutants

European journal of medicinal chemistry, 2021-01, Vol.209, p.112888-112888, Article 112888

2021

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New drug treatments for cystic fibrosis

BMJ (Online), 2020-01, Vol.368, p.m118

Smyth, Rosalind L

2020

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Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis

Cochrane database of systematic reviews, 2019-01, Vol.1 (1), p.CD009841

2019

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Measured fetal and neonatal exposure to Lumacaftor and Ivacaftor during pregnancy and while breastfeeding

Journal of cystic fibrosis, 2018-11, Vol.17 (6), p.779-782

2018

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The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis

Expert opinion on drug safety, 2017-11, Vol.16 (11), p.1305-1311

2017

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From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

Molecular biology of the cell, 2016-02, Vol.27 (3), p.424-433

2016

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Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

The Lancet (British edition), 2019-11, Vol.394 (10212), p.1940-1948

2019

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VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

The New England journal of medicine, 2018-10, Vol.379 (17), p.1612-1620

2018

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Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

The New England journal of medicine, 2019-11, Vol.381 (19), p.1809-1819

2019

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The pharmacokinetic interaction between ivacaftor and ritonavir in healthy volunteers

British journal of clinical pharmacology, 2017-10, Vol.83 (10), p.2235-2241

2017

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A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele

American journal of respiratory and critical care medicine, 2021-06, Vol.203 (12), p.1522-1532

2021

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The combination of tezacaftor and ivacaftor in the treatment of patients with cystic fibrosis: clinical evidence and future prospects in cystic fibrosis therapy

Therapeutic Advances in Respiratory Disease, 2019-04, Vol.13, p.1753466619844424

2019

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VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

The New England journal of medicine, 2018-10, Vol.379 (17), p.1599-1611

2018

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Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial

American journal of respiratory and critical care medicine, 2021-03, Vol.203 (5), p.585-593

2021

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Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

The lancet respiratory medicine, 2017-07, Vol.5 (7), p.557-567

2017

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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes

Structural identification of a hotspot on CFTR for potentiation

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

Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2–5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study

Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2–5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study

An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2–5 years (KLIMB)

From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis

Pharmacokinetic Enhancement of Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: A Cost Reduction Strategy to Address Global Disparities in Access

In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N‐of‐1 studies

Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis

1-BENZYLSPIRO[PIPERIDINE-4,1′-PYRIDO[3,4-b]indole] ‘co-potentiators’ for minimal function CFTR mutants

New drug treatments for cystic fibrosis

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis

Measured fetal and neonatal exposure to Lumacaftor and Ivacaftor during pregnancy and while breastfeeding

The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

The pharmacokinetic interaction between ivacaftor and ritonavir in healthy volunteers

A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele

The combination of tezacaftor and ivacaftor in the treatment of patients with cystic fibrosis: clinical evidence and future prospects in cystic fibrosis therapy

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial

Efficacy and safety of lumacaftor and ivacaftor in patients aged 6–11 years with cystic fibrosis homozygous for F508del-CFTR : a randomised, placebo-controlled phase 3 trial

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