PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.

[1]  J. Clancy,et al.  Evaluating the Impact of Stopping Chronic Therapies after Modulator Drug Therapy in Cystic Fibrosis: The SIMPLIFY Clinical Trial Study Design , 2021, Annals of the American Thoracic Society.

[2]  M. Parsek,et al.  Changes in fecal microbiota with CFTR modulator therapy: A pilot study. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[3]  S. Rowe,et al.  Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[4]  Samuel I. Miller,et al.  Maintenance tobramycin primarily affects untargeted bacteria in the CF sputum microbiome , 2020, Thorax.

[5]  J. Clancy,et al.  Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: Results from the prospect study. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[6]  Zhongyu Liu,et al.  Human Nasal Epithelial Organoids for Therapeutic Development in Cystic Fibrosis , 2020, Genes.

[7]  E. Mckone,et al.  Correlation between Ivacaftor-induced CFTR Activation in Airway Epithelial Cells and Improved Lung Function: A Proof-of-Concept. , 2020, Annals of the American Thoracic Society.

[8]  A. Mejias,et al.  Age and environmental exposures influence the fecal bacteriome of young children with cystic fibrosis , 2020, Pediatric pulmonology.

[9]  Samuel I. Miller,et al.  Fecal dysbiosis in infants with cystic fibrosis is associated with early linear growth failure , 2020, Nature Network Boston.

[10]  J. Harris,et al.  Changes in Airway Microbiome and Inflammation with Ivacaftor Treatment in Patients with Cystic Fibrosis and the G551D Mutation. , 2020, Annals of the American Thoracic Society.

[11]  S. Rowe,et al.  Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications. , 2019, American journal of respiratory and critical care medicine.

[12]  C. Teneback,et al.  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, The Lancet.

[13]  P. Lewindon,et al.  Accuracy of Transient Elastography Data Combined With APRI in Detection and Staging of Liver Disease in Pediatric Patients With Cystic Fibrosis. , 2019, Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association.

[14]  L. Lands,et al.  Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. , 2019, The New England journal of medicine.

[15]  Min Chang,et al.  Changes in APRI and FIB-4 in HBeAg-negative treatment-naive chronic hepatitis B patients with significant liver histological lesions receiving 5-year entecavir therapy , 2019, Clinical and Experimental Medicine.

[16]  R. Houwen,et al.  Cirrhosis associated with decreased survival and a 10-year lower median age at death of cystic fibrosis patients in the Netherlands. , 2019, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[17]  D. Waltz,et al.  Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles , 2019, ERJ Open Research.

[18]  P. Böelle,et al.  Cystic Fibrosis Liver Disease: Outcomes and Risk Factors in a Large Cohort of French Patients , 2018, Hepatology.

[19]  G. Sawicki,et al.  Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries. , 2020, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[20]  T. Corcoran,et al.  Effect of ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis patients with G551D-CFTR. , 2018, JCI insight.

[21]  M. Surette,et al.  Impact of CFTR modulation with Ivacaftor on Gut Microbiota and Intestinal Inflammation , 2018, Scientific Reports.

[22]  F. van Goor,et al.  VX‐445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles , 2018, The New England journal of medicine.

[23]  S. Rowe,et al.  Seeing cilia: imaging modalities for ciliary motion and clinical connections. , 2018, American journal of physiology. Lung cellular and molecular physiology.

[24]  M. Drumm,et al.  Cystic fibrosis-related diabetes is caused by islet loss and inflammation. , 2018, JCI insight.

[25]  G. Esmat,et al.  Changes in liver stiffness measurements and fibrosis scores following sofosbuvir based treatment regimens without interferon , 2017, Journal of gastroenterology and hepatology.

[26]  J. Jais,et al.  Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators , 2017, Scientific Reports.

[27]  E. Nash,et al.  The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers , 2017, Thorax.

[28]  S. Stanojevic,et al.  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. , 2017, The Lancet. Respiratory medicine.

[29]  L. Hoffman,et al.  Restoring Cystic Fibrosis Transmembrane Conductance Regulator Function Reduces Airway Bacteria and Inflammation in People with Cystic Fibrosis and Chronic Lung Infections , 2017, American journal of respiratory and critical care medicine.

[30]  Y. Chong,et al.  Circulating FGF19 closely correlates with bile acid synthesis and cholestasis in patients with primary biliary cirrhosis , 2017, PloS one.

[31]  J. Mainz,et al.  Abdominal symptoms in cystic fibrosis and their relation to genotype, history, clinical and laboratory findings , 2017, PloS one.

[32]  E. Tullis,et al.  Impact of CFTR Modulation on Intestinal pH, Motility, and Clinical Outcomes in Patients With Cystic Fibrosis and the G551D Mutation , 2017, Clinical and Translational Gastroenterology.

[33]  S. Stick,et al.  Sialic acid-to-urea ratio as a measure of airway surface hydration. , 2017, American journal of physiology. Lung cellular and molecular physiology.

[34]  B. Zemel,et al.  Trabecular and cortical bone deficits are present in children and adolescents with cystic fibrosis. , 2016, Bone.

[35]  Linbo Liu,et al.  Ion Channels and Transporters in Lung Function and Disease Combination therapy with cystic fibrosis transmembrane conductance regulator modulators augment the airway functional microanatomy , 2016 .

[36]  C. Minard,et al.  Aspartate aminotransferase to platelet ratio and fibrosis‐4 as biomarkers in biopsy‐validated pediatric cystic fibrosis liver disease , 2015, Hepatology.

[37]  S. Paranjape,et al.  Baseline Ultrasound and Clinical Correlates in Children with Cystic Fibrosis. , 2015, The Journal of pediatrics.

[38]  M. Karagas,et al.  Associations between Gut Microbial Colonization in Early Life and Respiratory Outcomes in Cystic Fibrosis. , 2015, The Journal of pediatrics.

[39]  M. Braddock,et al.  Targeting fibroblast growth factor 19 in liver disease: a potential biomarker and therapeutic target , 2015, Expert opinion on therapeutic targets.

[40]  B. Quon,et al.  A systematic review of factors associated with health-related quality of life in adolescents and adults with cystic fibrosis. , 2015, Annals of the American Thoracic Society.

[41]  B. Ramsey,et al.  Pseudomonas aeruginosa in cystic fibrosis patients with G551D-CFTR treated with ivacaftor. , 2015, Clinical infectious diseases : an official publication of the Infectious Diseases Society of America.

[42]  Umer Khan,et al.  Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis. , 2014, American journal of respiratory and critical care medicine.

[43]  Y. Sohma,et al.  Glucose-induced electrical activities and insulin secretion in pancreatic islet β-cells are modulated by CFTR , 2014, Nature Communications.

[44]  G. Clines,et al.  Cystic fibrosis-related bone disease: insights into a growing problem , 2013, Current opinion in endocrinology, diabetes, and obesity.

[45]  D. Borowitz,et al.  Intestinal complications of cystic fibrosis , 2013, Current opinion in pulmonary medicine.

[46]  P. Lee,et al.  Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. , 2013, The Lancet. Respiratory medicine.

[47]  M. Bellin,et al.  Insulin secretion improves in cystic fibrosis following ivacaftor correction of CFTR: a small pilot study , 2013, Pediatric diabetes.

[48]  G. Trimarchi,et al.  Stunting is an independent predictor of mortality in patients with cystic fibrosis. , 2013, Clinical nutrition.

[49]  M. Narkewicz,et al.  Cirrhosis and other liver disease in cystic fibrosis. , 2013, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[50]  H. Quinton,et al.  Better nutritional status in early childhood is associated with improved clinical outcomes and survival in patients with cystic fibrosis. , 2013, The Journal of pediatrics.

[51]  M. Hillman,et al.  Reduced levels of active GLP-1 in patients with cystic fibrosis with and without diabetes mellitus. , 2012, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

[52]  Matthias Griese,et al.  A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. , 2011, The New England journal of medicine.

[53]  P. Negulescu,et al.  Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809 , 2011, Proceedings of the National Academy of Sciences.

[54]  A. Baranova,et al.  Non-Invasive markers for hepatic fibrosis , 2011, BMC gastroenterology.

[55]  M. S. Kirkman,et al.  Epidemiology, Pathophysiology, and Prognostic Implications of Cystic Fibrosis–Related Diabetes , 2010, Diabetes Care.

[56]  J. Clancy,et al.  Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. , 2010, The New England journal of medicine.

[57]  P. Negulescu,et al.  Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770 , 2009, Proceedings of the National Academy of Sciences.

[58]  J. Kovář,et al.  Improved HPLC analysis of serum 7alpha-hydroxycholest-4-en-3-one, a marker of bile acid malabsorption. , 2008, Clinical chemistry.

[59]  T. Corcoran,et al.  Mucociliary clearance as an outcome measure for cystic fibrosis clinical research. , 2007, Proceedings of the American Thoracic Society.

[60]  D. Borowitz Update on the evaluation of pancreatic exocrine status in cystic fibrosis , 2005, Current opinion in pulmonary medicine.

[61]  J. Carlin,et al.  Association of body composition and lung function in children with cystic fibrosis , 2005, Pediatric pulmonology.