4.4 Review

New drugs in cystic fibrosis: what has changed in the last decade?

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Critical Care Medicine

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

Jane C. Davies et al.

Summary: The study demonstrates that ivacaftor is safe and effective in infants aged 4 to 12 months with cystic fibrosis and gating mutations, showing good results in terms of pharmacokinetics and safety in this age group.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2021)

Article Pharmacology & Pharmacy

Enhanced Expression of Human Epididymis Protein 4 (HE4) Reflecting Pro-Inflammatory Status Is Regulated by CFTR in Cystic Fibrosis Bronchial Epithelial Cells

Zsolt Bene et al.

Summary: In this study, it was found that decreased HE4 protein levels in CF patients treated with CFTR potentiator therapy were inversely correlated with lung function improvement. Modulation of CFTR function affected HE4 expression in CFBE cells, with activation of NF-kappa B pathway playing a role in regulating HE4 expression.

FRONTIERS IN PHARMACOLOGY (2021)

Article Health Care Sciences & Services

Personalized Medicine Based on Nasal Epithelial Cells: Comparative Studies with Rectal Biopsies and Intestinal Organoids

Iris A. L. Silva et al.

Summary: This study demonstrates that pHNE cells can serve as a model for evaluating the efficacy of CFTR modulator drugs for patients with CF and rare mutations, based on the correlation between CFTR basal function in pHNEs with rectal biopsies and CFTR rescue in pHNEs with intestinal organoids.

JOURNAL OF PERSONALIZED MEDICINE (2021)

Article Respiratory System

CFTR: New insights into structure and function and implications for modulation by small molecules

Bertrand Kleizen et al.

JOURNAL OF CYSTIC FIBROSIS (2020)

Review Pediatrics

Cystic fibrosis in the year 2020: A disease with a new face

Kris De Boeck

ACTA PAEDIATRICA (2020)

Review Pharmacology & Pharmacy

CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine

Miqueias Lopes-Pacheco

FRONTIERS IN PHARMACOLOGY (2020)

Review Health Policy & Services

A Review on the Use of Cystic Fibrosis Transmembrane Conductance Regulator Gene Modulators in Pediatric Patients

Michael Bitonti et al.

JOURNAL OF PEDIATRIC HEALTH CARE (2019)

Article Medicine, General & Internal

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

P. G. Middleton et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Article Physiology

Vx-809/Vx-770 treatment reduces inflammatory response to Pseudomonas aeruginosa in primary differentiated cystic fibrosis bronchial epithelial cells

Manon Ruffin et al.

AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY (2018)

Review Respiratory System

Cystic Fibrosis: Translating Molecular Mechanisms into Effective Therapies

Mark T. Jennings et al.

ANNALS OF THE AMERICAN THORACIC SOCIETY (2018)

Review Biochemistry & Molecular Biology

Role of CFTR in epithelial physiology

Vinciane Saint-Criq et al.

CELLULAR AND MOLECULAR LIFE SCIENCES (2017)

Article Medicine, General & Internal

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Steven M. Rowe et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Medicine, General & Internal

Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Jennifer L. Taylor-Cousar et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Review Pharmacology & Pharmacy

New horizons for cystic fibrosis treatment

Isabelle Fajac et al.

PHARMACOLOGY & THERAPEUTICS (2017)

Review Medicine, General & Internal

The changing epidemiology and demography of cystic fibrosis

Anne L. Stephenson et al.

PRESSE MEDICALE (2017)

Review Medicine, General & Internal

New treatments targeting the basic defects in cystic fibrosis

Isabelle Fajac et al.

PRESSE MEDICALE (2017)

Article Critical Care Medicine

Lumacaftor/Ivacaftor in Patients Aged 6-11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR

Carlos E. Milla et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2017)

Article Respiratory System

Epidemiology of Cystic Fibrosis

Kimberly A. Spoonhower et al.

CLINICS IN CHEST MEDICINE (2016)

Review Pharmacology & Pharmacy

CFTR Modulators: Shedding Light on Precision Medicine for Cystic Fibrosis

Miqueias Lopes-Pacheco

FRONTIERS IN PHARMACOLOGY (2016)

Review Critical Care Medicine

Progress in therapies for cystic fibrosis

Kris De Boeck et al.

LANCET RESPIRATORY MEDICINE (2016)

Article Medicine, General & Internal

Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

C. E. Wainwright et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

Review Pediatrics

Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors

George M. Solomon et al.

PEDIATRIC PULMONOLOGY (2015)

Review Pharmacology & Pharmacy

New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls

Scott C. Bell et al.

PHARMACOLOGY & THERAPEUTICS (2015)

Article Respiratory System

Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation

Kris De Boeck et al.

JOURNAL OF CYSTIC FIBROSIS (2014)

Article Critical Care Medicine

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with a G551D Mutation

Jane C. Davies et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2013)

Article Medicine, Research & Experimental

Physiology of Epithelial Chloride and Fluid Secretion

Raymond A. Frizzell et al.

COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2012)

Article Medicine, General & Internal

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

Bonnie W. Ramsey et al.

NEW ENGLAND JOURNAL OF MEDICINE (2011)

Article Medicine, General & Internal

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation

Frank J. Accurso et al.

NEW ENGLAND JOURNAL OF MEDICINE (2010)

Review Medicine, General & Internal

Cystic fibrosis

Brian P. O'Sullivan et al.

LANCET (2009)

Article Multidisciplinary Sciences

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

Fredrick Van Goor et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)