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Title
Cystic fibrosis in the year 2020: A disease with a new face
Authors
Keywords
-
Journal
ACTA PAEDIATRICA
Volume -, Issue -, Pages -
Publisher
Wiley
Online
2020-01-04
DOI
10.1111/apa.15155
References
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Note: Only part of the references are listed.- Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations
- (2019) Margarida D. Amaral et al. Journal of Cystic Fibrosis
- 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
- (2019) Harry G M Heijerman et al. LANCET
- Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
- (2019) Peter G. Middleton et al. NEW ENGLAND JOURNAL OF MEDICINE
- Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis
- (2019) Senne Cuyx et al. SEMINARS IN RESPIRATORY AND CRITICAL CARE MEDICINE
- Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
- (2019) Giulia Maule et al. Nature Communications
- Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR
- (2018) Jennifer L. Taylor-Cousar et al. Journal of Cystic Fibrosis
- WS01.2 Phase 2 initial results evaluating PTI-428, a novel CFTR amplifier, in patients with cystic fibrosis
- (2018) P. Flume et al. Journal of Cystic Fibrosis
- Lung function decline is delayed but not decreased in patients with cystic fibrosis and the R117H gene mutation
- (2018) Jeffrey S. Wagener et al. Journal of Cystic Fibrosis
- ECFS best practice guidelines: the 2018 revision
- (2018) Carlo Castellani et al. Journal of Cystic Fibrosis
- Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor
- (2018) Leona Bessonova et al. THORAX
- Ivacaftor treatment of cystic fibrosis in children aged 12 to
- (2018) Margaret Rosenfeld et al. Lancet Respiratory Medicine
- VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
- (2018) Dominic Keating et al. NEW ENGLAND JOURNAL OF MEDICINE
- VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles
- (2018) Jane C. Davies et al. NEW ENGLAND JOURNAL OF MEDICINE
- Modelling future trends in cystic fibrosis demography using the French Cystic Fibrosis Registry: update and sensitivity analysis
- (2017) Pierre-Régis Burgel et al. EUROPEAN RESPIRATORY JOURNAL
- The expansion and performance of national newborn screening programmes for cystic fibrosis in Europe
- (2017) Jürg Barben et al. Journal of Cystic Fibrosis
- WS13.3 Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: the international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF)
- (2017) M.W. Konstan et al. Journal of Cystic Fibrosis
- Ethnicity impacts the cystic fibrosis diagnosis: A note of caution
- (2017) Barbara Bosch et al. Journal of Cystic Fibrosis
- Bicarbonate in cystic fibrosis
- (2017) Karl Kunzelmann et al. Journal of Cystic Fibrosis
- The diagnosis of cystic fibrosis
- (2017) Kris De Boeck et al. PRESSE MEDICALE
- Equitable CF care as a basic human right
- (2016) Kris De Boeck et al. Journal of Cystic Fibrosis
- From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
- (2016) Gudio Veit et al. MOLECULAR BIOLOGY OF THE CELL
- Airway acidification initiates host defense abnormalities in cystic fibrosis mice
- (2016) V. S. Shah et al. SCIENCE
- Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
- (2016) Eric W F W Alton et al. THORAX
- 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
- (2016) Jane C Davies et al. Lancet Respiratory Medicine
- Newborn screening for cystic fibrosis
- (2016) Carlo Castellani et al. Lancet Respiratory Medicine
- Progress in therapies for cystic fibrosis
- (2016) Kris De Boeck et al. Lancet Respiratory Medicine
- Factors associated with response to treatment of pulmonary exacerbations in cystic fibrosis patients
- (2015) Valerie J. Waters et al. Journal of Cystic Fibrosis
- Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
- (2015) Claire E. Wainwright et al. NEW ENGLAND JOURNAL OF MEDICINE
- Treatment burden in patients with at least one class IV or VCFTRmutation
- (2015) Jonas Dewulf et al. PEDIATRIC PULMONOLOGY
- Outcomes of Infants With Indeterminate Diagnosis Detected by Cystic Fibrosis Newborn Screening
- (2015) C. L. Ren et al. PEDIATRICS
- Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial
- (2015) Richard B Moss et al. Lancet Respiratory Medicine
- The relative frequency of CFTR mutation classes in European patients with cystic fibrosis
- (2014) K. De Boeck et al. Journal of Cystic Fibrosis
- Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation
- (2014) Kris De Boeck et al. Journal of Cystic Fibrosis
- Key findings of the US Cystic Fibrosis Foundation's clinical practice benchmarking project
- (2014) Michael P Boyle et al. BMJ Quality & Safety
- Factors associated with FEV1 decline in cystic fibrosis: analysis of the ECFS Patient Registry
- (2013) E. Kerem et al. EUROPEAN RESPIRATORY JOURNAL
- A functional CFTR assay using primary cystic fibrosis intestinal organoids
- (2013) Johanna F Dekkers et al. NATURE MEDICINE
- Ageing in Cystic Fibrosis and Long-term Survival
- (2013) N.J. Simmonds Paediatric Respiratory Reviews
- Effect of Estrogen on Pseudomonas Mucoidy and Exacerbations in Cystic Fibrosis
- (2012) Sanjay H. Chotirmall et al. NEW ENGLAND JOURNAL OF MEDICINE
- Improved survival in cystic fibrosis patients diagnosed by newborn screening compared to a historical cohort from the same centre
- (2011) F. N. Dijk et al. ARCHIVES OF DISEASE IN CHILDHOOD
- A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation
- (2011) Bonnie W. Ramsey et al. NEW ENGLAND JOURNAL OF MEDICINE
- Progression of early structural lung disease in young children with cystic fibrosis assessed using CT
- (2011) Lauren S Mott et al. THORAX
- Cystic fibrosis across Europe: EuroCareCF analysis of demographic data from 35 countries
- (2010) Gita Mehta et al. Journal of Cystic Fibrosis
- Year-to-year changes in lung function in individuals with cystic fibrosis
- (2010) Theodore G. Liou et al. Journal of Cystic Fibrosis
- Early referral to cystic fibrosis specialist centre impacts on respiratory outcome
- (2008) P. Lebecque et al. Journal of Cystic Fibrosis
- Cystic fibrosis: impaired bicarbonate secretion and mucoviscidosis
- (2008) Paul M Quinton LANCET
- Time Trends in Birth Incidence of Cystic Fibrosis in Two European Areas: Data from Newborn Screening Programs
- (2007) Virginie Scotet et al. JOURNAL OF PEDIATRICS
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