PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.
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L. Hoffman | M. Rosenfeld | J. Clancy | S. Freedman | S. Donaldson | F. Ratjen | S. Rowe | Pradeep K. Singh | S. Schwarzenberg | G. Solomon | M. Narkewicz | S. Sagel | D. Nichols | J. Pittman | D. Gelfond | M. Stalvey | J. VanDalfsen | S. Kirby | C. Frederick | A. Kelly | Jessica E. Pittman | J. Vandalfsen
[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.