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。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.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2021)
Reduction of pulmonary exacerbations in young children with cystic fibrosis during the COVID-19 pandemic
Shreya Patel et al.
PEDIATRIC PULMONOLOGY (2021)
Magnetic Resonance Imaging Detects Progression of Lung Disease and Impact of Newborn Screening in Preschool Children with Cystic Fibrosis
Mirjam Stahl et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2021)
The future of cystic fibrosis care: a global perspective
Scott C. Bell et al.
LANCET RESPIRATORY MEDICINE (2020)
Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications
Marcus A. Mall et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2020)
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
John McNamara et al.
LANCET RESPIRATORY MEDICINE (2019)
Growth failure and treatment in cystic fibrosis
Trang N. Le et al.
JOURNAL OF CYSTIC FIBROSIS (2019)
Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study
Margaret Rosenfeld et al.
LANCET RESPIRATORY MEDICINE (2018)
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
Felix Ratjen et al.
LANCET RESPIRATORY MEDICINE (2017)
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study
Michael W. Konstan et al.
LANCET RESPIRATORY MEDICINE (2017)
Early Lung Disease in Infants and Preschool Children with Cystic Fibrosis What Have We Learned and What Should We Do about It?
Sarath C. Ranganathan et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2017)
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)
Cystic fibrosis in young children: A review of disease manifestation, progression, and response to early treatment
Donald R. VanDevanter et al.
JOURNAL OF CYSTIC FIBROSIS (2016)
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
Jane C. Davies et al.
LANCET RESPIRATORY MEDICINE (2016)
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
C. E. Wainwright et al.
NEW ENGLAND JOURNAL OF MEDICINE (2015)
Sweat chloride as a biomarker of CFTR activity: Proof of concept and ivacaftor clinical trial data
Frank J. Accurso et al.
JOURNAL OF CYSTIC FIBROSIS (2014)
Evolution of lung function during the first year of life in newborn screened cystic fibrosis infants
The Thanh-Diem Nguyen et al.
THORAX (2014)
A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect
Michael P. Boyle et al.
LANCET RESPIRATORY MEDICINE (2013)
Pancreatic Damage in Fetal and Newborn Cystic Fibrosis Pigs Involves the Activation of Inflammatory and Remodeling Pathways
Maisam Abu-El-Haija et al.
AMERICAN JOURNAL OF PATHOLOGY (2012)
Lung function is abnormal in 3-month-old infants with cystic fibrosis diagnosed by newborn screening
Ah-Fong Hoo et al.
THORAX (2012)
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)
Mutations in the cystic fibrosis transmembrane regulator gene and in vivo transepithelial potentials
Michael Wilschanski et al.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2006)
Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules
F Van Goor et al.
AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY (2006)
Early decline of pancreatic function in cystic fibrosis patients with class 1 or 2 CFTR mutations
T Walkowiak et al.
JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION (2005)
Growth and nutritional indexes in early life predict pulmonary function in cystic fibrosis
MW Konstan et al.
JOURNAL OF PEDIATRICS (2003)
分享论文
