Cited by 685Cystic Fibrosis Research AdvancesGenetic and Kidney Cyst DiseasesNeonatal Respiratory Health Research

The DOI matches its Crossref record; 194 of the 195 references it cites were found in a registry or an index.

Cystic fibrosis

Open access

Shteinberg, Michal, Haq, Iram J, Polineni, Deepika, Davies, Jane C·2021·The Lancet

Open this paper in CoChat — free account

Abstract

Cystic fibrosis is a monogenic disease considered to affect at least 100 000 people worldwide. Mutations in CFTR, the gene encoding the epithelial ion channel that normally transports chloride and bicarbonate, lead to impaired mucus hydration and clearance. Classical cystic fibrosis is thus characterised by chronic pulmonary infection and inflammation, pancreatic exocrine insufficiency, male infertility, and might include several comorbidities such as cystic fibrosis-related diabetes or cystic fibrosis liver disease. This autosomal recessive disease is diagnosed in many regions following newborn screening, whereas in other regions, diagnosis is based on a group of recognised multiorgan clinical manifestations, raised sweat chloride concentrations, or CFTR mutations. Disease that is less easily diagnosed, and in some cases affecting only one organ, can be seen in the context of gene variants leading to residual protein function. Management strategies, including augmenting mucociliary clearance and aggressively treating infections, have gradually improved life expectancy for people with cystic fibrosis. However, restoration of CFTR function via new small molecule modulator drugs is transforming the disease for many patients. Clinical trial pipelines are actively exploring many other approaches, which will be increasingly needed as survival improves and as the population of adults with cystic fibrosis increases. Here, we present the current understanding of CFTR mutations, protein function, and disease pathophysiology, consider strengths and limitations of current management strategies, and look to the future of multidisciplinary care for those with cystic fibrosis.

Integrity signals

Registry

Crossref · 10.1016/s0140-6736(20)32542-3

Cites

194 of 194 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Claims the paper makes

We couldn’t retrieve this paper’s full text, so its claims and self-check weren’t run. The registry and citation checks still apply.

Standardisation of spirometry
Miller, M. R., Hankinson, J., Brusasco, V., Burgos, F., Casaburi, R., Coates, A., Crapo, R., Enright, P., van der Grinten, C. P. M., Gustafsson, P., et al. (2005) · European Respiratory Journal · doi:10.1183/09031936.05.00034805 · Cited by 15,627
Identification of the Cystic Fibrosis Gene: Genetic Analysis
Kerem, B.-S., Rommens, J. M., Buchanan, J. A., Markiewicz, D., Cox, T. K., Chakravarti, A., Buchwald, M., & Tsui, L.-C. (1989) · Science · doi:10.1126/science.2570460 · Cited by 4,142
Antibiotic resistance in Pseudomonas aeruginosa: mechanisms and alternative therapeutic strategies
Pang, Z., Raudonis, R., Glick, B. R., Lin, T., & Cheng, Z. (2018) · Biotechnology Advances · doi:10.1016/j.biotechadv.2018.11.013 · Cited by 2,520
Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele
Middleton, P. G., Mall, M. A., Dřevínek, P., Lands, L. C., McKone, E. F., Polineni, D., Ramsey, B. W., Taylor-Cousar, J. L., Tullis, E., Vermeulen, F., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1908639 · Cited by 2,198
Self-citation
A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
Ramsey, B. W., Davies, J., McElvaney, N. G., Tullis, E., Bell, S. C., Dřevínek, P., Griese, M., McKone, E. F., Wainwright, C. E., Konstan, M. W., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1105185 · Cited by 2,195
Self-citation
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Wainwright, C. E., Elborn, J. S., Ramsey, B. W., Marigowda, G., Huang, X., Cipolli, M., Colombo, C., Davies, J. C., De Boeck, K., Flume, P. A., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1409547 · Cited by 1,622
Self-citation
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
Heijerman, H. G. M., McKone, E. F., Downey, D. G., Van Braeckel, E., Rowe, S. M., Tullis, E., Mall, M. A., Welter, J. J., Ramsey, B. W., McKee, C. M., et al. (2019) · The Lancet · doi:10.1016/s0140-6736(19)32597-8 · Cited by 1,414
Intermittent Administration of Inhaled Tobramycin in Patients with Cystic Fibrosis
Ramsey, B. W., PEPE, M. S., Quan, J. M., Otto, K., Montgomery, A. B., Williams‐Warren, J., Vasiljev-K, M., Borowitz, D., Bowman, C. M., Marshall, B. C., et al. (1999) · New England Journal of Medicine · doi:10.1056/nejm199901073400104 · Cited by 1,296
The International Thoracic Organ Transplant Registry of the International Society for Heart and Lung Transplantation: Thirty-sixth adult lung and heart–lung transplantation Report—2019; Focus theme: donor and recipient size match
Chambers, D. C., Cherikh, W. S., Harhay, M. O., Hayes, D., Hsich, E., Khush, K. K., Meiser, B., Potena, L., Rossano, J. W., Toll, A. E., et al. (2019) · The Journal of Heart and Lung Transplantation · doi:10.1016/j.healun.2019.08.001 · Cited by 1,269
The future of cystic fibrosis care: a global perspective
Bell, S. C., Mall, M. A., Gutierrez, H., Macek, M., Madge, S. L., Davies, J. C., Burgel, P., Tullis, E., Castaños, C., Castellani, C., et al. (2019) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(19)30337-6 · Cited by 1,081
Self-citation
A functional CFTR assay using primary cystic fibrosis intestinal organoids
Dekkers, J. F., Wiegerinck, C. L., de Jonge, H. R., Bronsveld, I., Janssens, H. M., de Winter-de Groot, K. M., Brandsma, A. M., de Jong, N. W. M., Bijvelds, M. J. C., Scholte, B. J., et al. (2013) · Nature Medicine · doi:10.1038/nm.3201 · Cited by 999
Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation
Farrell, P. M., White, T. B., Ren, C. L., Hempstead, S. E., Accurso, F., Derichs, N., Howenstine, M., McColley, S. A., Rock, M., Rosenfeld, M., et al. (2017) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2016.09.064 · Cited by 953
A Periciliary Brush Promotes the Lung Health by Separating the Mucus Layer from Airway Epithelia
Button, B. M., Cai, L.-H., Ehré, C., Kesımer, M., Hill, D. B., Sheehan, J. K., Boucher, R. C., & Rubinstein, M. (2012) · Science · doi:10.1126/science.1223012 · Cited by 906
Reduced airway surface pH impairs bacterial killing in the porcine cystic fibrosis lung
Pezzulo, A. A., Tang, X. X., Hoegger, M. J., Alaiwa, M. H. A., Ramachandran, S. S., Moninger, T. O., Karp, P. H., Wohlford-Lenane, C., Haagsman, H. P., van Eijk, M., et al. (2012) · Nature · doi:10.1038/nature11130 · Cited by 831
Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del
Taylor-Cousar, J. L., Munck, A., McKone, E. F., van der Ent, C. K., Moeller, A., Simard, C., Wang, L. T., Ingenito, E. P., McKee, C., Lu, Y., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1709846 · Cited by 794
British Thoracic Society guidelines for the management of non-tuberculous mycobacterial pulmonary disease (NTM-PD)
HAWORTH, C. S., Banks, J., Capstick, T., Fisher, A. J., Gorsuch, T., Laurenson, I. F., Leitch, A., Loebinger, M. R., Milburn, H. J., Nightingale, M., et al. (2017) · Thorax · doi:10.1136/thoraxjnl-2017-210927 · Cited by 791
Involvement of Nitric Oxide in Biofilm Dispersal of Pseudomonas aeruginosa
Barraud, N., Hassett, D. J., Hwang, S.-H., Rice, S. A., Kjelleberg, S. J., & Webb, J. S. (2006) · Journal of Bacteriology · doi:10.1128/jb.00779-06 · Cited by 785
ECFS best practice guidelines: the 2018 revision
Castellani, C., Duff, A. J. A., Bell, S. C., Heijerman, H. G. M., Munck, A., Ratjen, F., Sermet-Gaudelus, I., Southern, K. W., Barben, J., Flume, P. A., et al. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.02.006 · Cited by 765
CYSTIC FIBROSIS: GENOTYPIC AND PHENOTYPIC VARIATIONS
Zielenski, J., & Tsui, L. (1995) · Annual Review of Genetics · doi:10.1146/annurev.ge.29.120195.004021 · Cited by 651
New concepts of the pathogenesis of cystic fibrosis lung disease
Boucher, R. C. (2003) · European Respiratory Journal · doi:10.1183/09031936.03.00057003 · Cited by 645
From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
Veit, G., Avramescu, R. G., Chiang, A., Houck, S. A., Cai, Z., Peters, K. W., Hong, J. S., Pollard, H. B., Guggino, W. B., Balch, W. E., et al. (2016) · Molecular Biology of the Cell · doi:10.1091/mbc.e14-04-0935 · Cited by 613
Risk Factors for Bronchiectasis in Children with Cystic Fibrosis
Sly, P. D., Gangell, C. L., Chen, L., Ware, R. S., Ranganathan, S., Mott, L. S., Murray, C. P. J., & Stick, S. M. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1301725 · Cited by 596
Lung Disease at Diagnosis in Infants with Cystic Fibrosis Detected by Newborn Screening
Sly, P. D., Brennan, S., Gangell, C. L., de Klerk, N. H., Murray, C. P. J., Mott, L. S., Stick, S. M., Robinson, P. J., ROBERTSON, C. F., & Ranganathan, S. (2009) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.200901-0069oc · Cited by 552
ESPEN-ESPGHAN-ECFS guidelines on nutrition care for infants, children, and adults with cystic fibrosis
Turck, D., Braegger, C., Colombo, C., Declercq, D., Morton, A. M., Pancheva, R., Robberecht, E., Stern, M., Strandvik, B., Wolfe, S., et al. (2016) · Clinical Nutrition · doi:10.1016/j.clnu.2016.03.004 · Cited by 546
Prevalence of depression and anxiety in patients with cystic fibrosis and parent caregivers: results of The International Depression Epidemiological Study across nine countries
Quittner, A. L., Goldbeck, L., Abbott, J., Duff, A., Lambrecht, P., Solé, A., Tibosch, M. M., Bergsten Brucefors, A., Yüksel, H., Catastini, P., et al. (2014) · Thorax · doi:10.1136/thoraxjnl-2014-205983 · Cited by 524
Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis
Rowe, S. M., Daines, C., Ringshausen, F. C., Kerem, E., Wilson, J., Tullis, E., Nair, N., Simard, C., Han, L., Ingenito, E. P., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1709847 · Cited by 522
Self-citation
Effect of long term treatment with azithromycin on disease parameters in cystic fibrosis: a randomised trial
Wolter, J. M. (2002) · Thorax · doi:10.1136/thorax.57.3.212 · Cited by 516
Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation
Clancy, J. P., Rowe, S. M., Accurso, F. J., Aitken, M. L., Amin, R. S., Ashlock, M. A., Ballmann, M., Boyle, M., Bronsveld, I., Campbell, P. W., et al. (2011) · Thorax · doi:10.1136/thoraxjnl-2011-200393 · Cited by 514
Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis
Rowe, S. M., Heltshe, S. L., Gonska, T., Donaldson, S. H., Borowitz, D., Gelfond, D., Sagel, S. D., Khan, U., Mayer-Hamblett, N., Van Dalfsen, J. M., et al. (2014) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201404-0703oc · Cited by 492
International Committee on Mental Health in Cystic Fibrosis: Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus statements for screening and treating depression and anxiety.
Quittner, A. L., Abbott, J., Georgiopoulos, A. M., Goldbeck, L., Smith, B., Hempstead, S. E., Marshall, B., Sabadosa, K. A., & Elborn, S. (2016) · Thorax · doi:10.1136/thoraxjnl-2015-207488 · Cited by 465
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
Alton, E. W. F. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., Boyd, A. C., Brand, J., Buchan, R., Calcedo, R., et al. (2015) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(15)00245-3 · Cited by 428
Self-citation
Best practice guidance for the diagnosis and management of cystic fibrosis-associated liver disease
Debray, D., Kelly, D. A., Houwen, R. H. J., Strandvik, B., & Colombo, C. (2011) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(11)60006-4 · Cited by 428
Recommendations for the classification of diseases as CFTR-related disorders
Bombieri, C., Claustres, M., Boeck, K. D., Derichs, N., DODGE, J. A., Girodon, E., Sermet‐Gaudelus, I., Schwarz, M. J., Tzetis, M., Wilschanski, M. A., et al. (2011) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(11)60014-3 · Cited by 425
Infection Prevention and Control Guideline for Cystic Fibrosis: 2013 Update
Saiman, L. M., Siegel, J. D., LiPuma, J. J., Brown, R. F., Bryson, E. A., Chambers, M. J., Downer, V. S., Fliege, J. D., Hazle, L., Jain, M., et al. (2014) · Infection Control and Hospital Epidemiology · doi:10.1086/676882 · Cited by 405
Restoring Cystic Fibrosis Transmembrane Conductance Regulator Function Reduces Airway Bacteria and Inflammation in People with Cystic Fibrosis and Chronic Lung Infections
Hisert, K. B., Heltshe, S. L., Pope, C. E., Jorth, P. A., Wu, X., Edwards, R. M., Radey, M. C., Accurso, F. J., Wolter, D. J., Cooke, G., et al. (2017) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201609-1954oc · Cited by 396
Amikacin Liposome Inhalation Suspension for Treatment-Refractory Lung Disease Caused by Mycobacterium avium Complex (CONVERT). A Prospective, Open-Label, Randomized Study
Griffith, D. E., Eagle, G. L., Thomson, R. M., Aksamit, T. R., Hasegawa, N., Morimoto, K., Addrizzo-Harris, D. J., O’Donnell, A. E., Marras, T. K., Flume, P. A., et al. (2018) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201807-1318oc · Cited by 362
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
Davies, J. C., Cunningham, S., Harris, W. T., Lapey, A., Regelmann, W. E., Sawicki, G. S., Southern, K. W., Robertson, S. M. C., Green, Y., Cooke, J., et al. (2016) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(15)00545-7 · Cited by 354
Self-citation
Bicarbonate and functional CFTR channel are required for proper mucin secretion and link cystic fibrosis with its mucus phenotype
Gustafsson, J. K., Ermund, A., Ambort, D., Johansson, M., Nilsson, H. E., Thorell, K., Hebert, H., Sjövall, H., & Hansson, G. C. (2012) · The Journal of Experimental Medicine · doi:10.1084/jem.20120562 · Cited by 345
Cystic Fibrosis Pulmonary Guidelines: Airway Clearance Therapies
Flume, P. A., Robinson, K. A., O'Sullivan, B. P., Finder, J. D., Vender, R. L., Willey-Courand, D.-B., White, T. B., & Marshall, B. C. (2009) · Respiratory Care · doi:10.4187/respcare.09540522 · Cited by 340
Effective mucus clearance is essential for respiratory health
Randell, S. H., & Boucher, R. C. (2006) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2006-0082sf · Cited by 335
Bronchiectasis in Infants and Preschool Children Diagnosed with Cystic Fibrosis after Newborn Screening
Stick, S. M., Brennan, S., Murray, C. P. J., Douglas, T. A., von Ungern-Sternberg, B. S., Garratt, L. W., Gangell, C. L., de Klerk, N. H., Linnane, B. M., Ranganathan, S., et al. (2009) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2009.05.005 · Cited by 333
Effect of Azithromycin on Pulmonary Function in Patients With Cystic Fibrosis Uninfected With Pseudomonas aeruginosa A Randomized Controlled Trial
Saiman, L. M. (2010) · JAMA · doi:10.1001/jama.2010.563 · Cited by 326
Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis
Hewer, S. C. L., & Smyth, A. R. (2014) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd004197.pub4 · Cited by 324
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
Ratjen, F. A., Hug, C., Marigowda, G., Tian, S., Huang, X., Stanojevic, S., Milla, C. E., Robinson, P. D., Waltz, D. A., Davies, J. C., et al. (2017) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(17)30215-1 · Cited by 297
Self-citation
Future trends in cystic fibrosis demography in 34 European countries
Burgel, P., Bellis, G., Olesen, H. V., Viviani, L., Zolin, A., Blasi, F., & Elborn, J. S. (2015) · European Respiratory Journal · doi:10.1183/09031936.00196314 · Cited by 293
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
Konstan, M. W., McKone, E. F., Moss, R. B., Marigowda, G., Tian, S., Waltz, D., Huang, X., Lubarsky, B., Rubin, J., Millar, S. J., et al. (2017) · The Lancet Respiratory Medicine · doi:10.1016/s2213-2600(16)30427-1 · Cited by 259
Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries
Volkova, N., Moy, K., Evans, J., Campbell, D., Tian, S., Simard, C., Higgins, M., Konstan, M. W., Sawicki, G. S., Elbert, A., et al. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.05.015 · Cited by 258
The Global Lung Function Initiative (GLI) Network: bringing the world’s respiratory reference values together
Cooper, B. G., Stocks, J., Hall, G. L., Culver, B. H., Steenbruggen, I., Carter, K. W., Thompson, B. R., Graham, B. L., Miller, M. R., Ruppel, G. L., et al. (2017) · Breathe · doi:10.1183/20734735.012717 · Cited by 256
Type of CFTR Mutation Determines Risk of Pancreatitis in Patients With Cystic Fibrosis
Ooi, C. Y., Dorfman, R., Cipolli, M., Gonska, T. Y., Castellani, C., Keenan, K., Freedman, S. D., Zielenski, J., Berthiaume, Y., Corey, M. L., et al. (2010) · Gastroenterology · doi:10.1053/j.gastro.2010.09.046 · Cited by 255
Epidemiology, Biology, and Impact of Clonal Pseudomonas aeruginosa Infections in Cystic Fibrosis
Parkins, M. D., Somayaji, R., & Waters, V. J. (2018) · Clinical Microbiology Reviews · doi:10.1128/cmr.00019-18 · Cited by 252
Lung transplant referral for individuals with cystic fibrosis: cystic Fibrosis Foundation consensus guidelines
Ramos, K. J., Smith, P. J., McKone, E. F., Pilewski, J. M., Lucy, A., Hempstead, S. E., Tallarico, E., Faro, A., Rosenbluth, D. B., Gray, A. L., et al. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.03.002 · Cited by 227
Vitamin D supplementation for cystic fibrosis
Ferguson, J. H., & Chang, A. B. (2014) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd007298.pub4 · Cited by 219
Phage therapy is highly effective against chronic lung infections withPseudomonas aeruginosa
Waters, E. M., Neill, D. R., Kaman, B., Sahota, J. S., Clokie, M. R. J., Winstanley, C., & Kadioglu, A. (2017) · Thorax · doi:10.1136/thoraxjnl-2016-209265 · Cited by 216
Comparison of Lung Clearance Index and Magnetic Resonance Imaging for Assessment of Lung Disease in Children with Cystic Fibrosis
Stahl, M., Wielpütz, M. O., Graeber, S. Y., Joachim, C., Sommerburg, O., Kauczor, H., Puderbach, M., Eichinger, M., & Mall, M. A. (2016) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201604-0893oc · Cited by 213
Guidelines for the diagnosis and management of distal intestinal obstruction syndrome in cystic fibrosis patients
Colombo, C., Ellemunter, H., Houwen, R. H. J., Munck, À., Taylor, C., & Wilschanski, M. A. (2011) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(11)60005-2 · Cited by 198
Mucus accumulation in the lungs precedes structural changes and infection in children with cystic fibrosis
Esther, C. R., Muhlebach, M. S., Ehré, C., Hill, D. B., Wolfgang, M. C., Kesımer, M., Ramsey, K. A., Markovetz, M. R., Garbarine, I. C., Forest, M. G., et al. (2019) · Science Translational Medicine · doi:10.1126/scitranslmed.aav3488 · Cited by 198
Cystic fibrosis carriers are at increased risk for a wide range of cystic fibrosis-related conditions
Miller, A. C., Comellas, A. P., Hornick, D. B., Stoltz, D. A., Cavanaugh, J. E., Gerke, A. K., Welsh, M. J., Zabner, J., & Polgreen, P. M. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1914912117 · Cited by 198
Cystic fibrosis colorectal cancer screening consensus recommendations
Hadjiliadis, D., Khoruts, A., Zauber, A. G., Hempstead, S. E., Maisonneuve, P., Lowenfels, A. B., Braid, A. L., Cullina, J., Daggett, A., Fink, A., et al. (2018) · Gastroenterology · doi:10.1053/j.gastro.2017.12.012 · Cited by 195
In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis
Sun, X., Yi, Y., Yan, Z., Rosen, B. H., Liang, B., Winter, M. C., Evans, T. I. A., Rotti, P. G., Yu, Y., Gray, J. S., et al. (2019) · Science Translational Medicine · doi:10.1126/scitranslmed.aau7531 · Cited by 193
Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations
Nudelman, I., Rebibo-Sabbah, A., Cherniavsky, M., Белахов, В. В., Hainrichson, M., Chen, F., Schacht, J., Pilch, D. S., Ben‐Yosef, T., & Baasov, T. (2009) · Journal of Medicinal Chemistry · doi:10.1021/jm801640k · Cited by 190
Cystic fibrosis–related diabetes is caused by islet loss and inflammation
Hart, N. J., Aramandla, R., Poffenberger, G., Fayolle, C., Thames, A. H., Bautista, A., Spigelman, A. F., Babon, J. A. B., DeNicola, M., Dadi, P. K., et al. (2018) · JCI Insight · doi:10.1172/jci.insight.98240 · Cited by 183
Molecular Basis of Azithromycin-Resistant Pseudomonas aeruginosa Biofilms
Gillis, R. J., White, K. G., Choi, K., Wagner, V., Schweizer, H. P., & Iglewski, B. H. (2005) · Antimicrobial Agents and Chemotherapy · doi:10.1128/aac.49.9.3858-3867.2005 · Cited by 183
Risk of gastrointestinal cancers in patients with cystic fibrosis: a systematic review and meta-analysis
Yamada, A., Komaki, Y., Komaki, F., Micić, D., Zullow, S., & Sakuraba, A. (2018) · The Lancet Oncology · doi:10.1016/s1470-2045(18)30188-8 · Cited by 182
Cystic Fibrosis Screen Positive, Inconclusive Diagnosis (CFSPID): A new designation and management recommendations for infants with an inconclusive diagnosis following newborn screening
Munck, À., Mayell, S. J., Winters, V., Shawcross, A., Derichs, N., Parad, R. B., Barben, J., & Southern, K. W. (2015) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2015.01.001 · Cited by 173
Overcoming Drug Resistance with Alginate Oligosaccharides Able To Potentiate the Action of Selected Antibiotics
Khan, S. S., Tøndervik, A., Sletta, H., Klinkenberg, G., Emanuel, C., Onsøyen, E., Myrvold, R., Howe, R. A., Walsh, T. R., Hill, K. E., et al. (2012) · Antimicrobial Agents and Chemotherapy · doi:10.1128/aac.00525-12 · Cited by 172
ISPAD Clinical Practice Consensus Guidelines 2018: management of cystic fibrosis-related diabetes in children and adolescents
Moran, A., Pillay, K., Becker, D., Granados, A., Hameed, S., & Acerini, C. L. (2018) · Pediatric Diabetes · doi:10.1111/pedi.12732 · Cited by 171
Cystic Fibrosis Foundation consensus guidelines for the care of individuals with advanced cystic fibrosis lung disease
Kapnadak, S. G., DiMango, E., Hadjiliadis, D., Hempstead, S. E., Tallarico, E., Pilewski, J. M., Faro, A., Albright, J. T., Benden, C., Blair, S., et al. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.02.015 · Cited by 167
Pseudomonas aeruginosa infection in cystic fibrosis: pathophysiological mechanisms and therapeutic approaches
Lund‐Palau, H., Turnbull, A. R., Bush, A., Bardin, E., Cameron, L. P., Soren, O., Wierre-Gore, N., Alton, E. W. F. W., Bundy, J. G., Connett, G. J., et al. (2016) · Expert Review of Respiratory Medicine · doi:10.1080/17476348.2016.1177460 · Cited by 165
Self-citation
Meconium ileus in Cystic Fibrosis
Sathe, M., & Houwen, R. H. J. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.06.007 · Cited by 165
Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor
Xue, X., Mutyam, V., Tang, L., Biswas, S., Du, M., Jackson, L. A., Dai, Y., Белахов, В. В., Shalev, M., Chen, F., et al. (2013) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2013-0282oc · Cited by 161
The Cystic Fibrosis of Exocrine PancreasPreprint
Wilschanski, M. A., & Novak, I. (2013) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a009746 · Cited by 160
Ursodeoxycholic acid for cystic fibrosis-related liver disease
Cheng, K., Ashby, D., & Smyth, R. L. (2017) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd000222.pub4 · Cited by 158
Intestinal current measurement for diagnostic classification of patients with questionable cystic fibrosis: validation and reference data
Derichs, N., Sanz, J., von Känel, T., Stolpe, C., Zapf, A., Tümmler, B., Gallati, S., & Ballmann, M. (2010) · Thorax · doi:10.1136/thx.2009.125088 · Cited by 144
A Functional Anatomic Defect of the Cystic Fibrosis Airway
Birket, S. E., Chu, K. K., Liu, L., Houser, G. H., Diephuis, B. J., Wilsterman, E. J., Dierksen, G. A., Mazur, M., Shastry, S., Li, Y., et al. (2014) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201404-0670oc · Cited by 143
Co-encapsulation of gallium with gentamicin in liposomes enhances antimicrobial activity of gentamicin against Pseudomonas aeruginosa
Halwani, M., Yebio, B., Suntres, Z. E., Alipour, M., Azghani, A. O., & Omri, A. (2008) · Journal of Antimicrobial Chemotherapy · doi:10.1093/jac/dkn422 · Cited by 140
Features of Severe Liver Disease With Portal Hypertension in Patients With Cystic Fibrosis
Stonebraker, J. R., Ooi, C. Y., Pace, R. G., Corvol, H., Knowles, M. R., Durie, P. R., & Ling, S. C. (2016) · Clinical Gastroenterology and Hepatology · doi:10.1016/j.cgh.2016.03.041 · Cited by 138
ERS/TSANZ Task Force Statement on the management of reproduction and pregnancy in women with airways diseases
Middleton, P. G., Gade, E. J., Aguilera, C., Mackillop, L. H., Button, B., Coleman, C., Johnson, B. A., Albrechtsen, C. K., Edenborough, F. P., Comas, D. R., et al. (2019) · European Respiratory Journal · doi:10.1183/13993003.01208-2019 · Cited by 138
A multinational report to characterise SARS-CoV-2 infection in people with cystic fibrosis
Cosgriff, R., Ahern, S., Bell, S. C., Brownlee, K. G., Burgel, P., Byrnes, C., Corvol, H., Cheng, S. Y., Elbert, A., Faro, A., et al. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.04.012 · Cited by 138
Cystic Fibrosis Transmembrane Conductance Regulator-Related Metabolic Syndrome and Cystic Fibrosis Screen Positive, Inconclusive Diagnosis
Ren, C. L., Borowitz, D., Gonska, T. Y., Howenstine, M. S., Levy, H., Massie, J., Milla, C. E., Munck, À., & Southern, K. W. (2017) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2016.09.066 · Cited by 132
Infection Is Not Required for Mucoinflammatory Lung Disease in CFTR-Knockout Ferrets
Rosen, B. H., Evans, T. I. A., Moll, S. R., Gray, J. S., Liang, B., Sun, X., Zhang, Y., Jensen-Cody, C. W., Swatek, A. M., Zhou, W., et al. (2018) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201708-1616oc · Cited by 132
Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine
Pranke, I. M., Golec, A., Hinzpeter, A., Edelman, A., & Sermet‐Gaudelus, I. (2019) · Frontiers in Pharmacology · doi:10.3389/fphar.2019.00121 · Cited by 127
Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis
Hewer, S. C. L., & Smyth, A. R. (2017) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd004197.pub5 · Cited by 127
Report of the European Respiratory Society/European Cystic Fibrosis Society task force on the care of adults with cystic fibrosis
Elborn, J. S., Bell, S. C., Madge, S. L., Burgel, P., Castellani, C., Conway, S., Rijcke, K. D., Dembski, B., Dřevı́nek, P., Heijerman, H. G. M., et al. (2015) · European Respiratory Journal · doi:10.1183/13993003.00592-2015 · Cited by 122
Diagnosis of Cystic Fibrosis in Screened Populations
Farrell, P. M., White, T. B., Howenstine, M. S., Munck, À., Parad, R. B., Rosenfeld, M., Sommerburg, O., Accurso, F. J., Davies, J. C., Rock, M. J., et al. (2017) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2016.09.065 · Cited by 121
Self-citation
Antimicrobial susceptibility testing (AST) and associated clinical outcomes in individuals with cystic fibrosis: A systematic review
Somayaji, R., Parkins, M. D., Shah, A., Martiniano, S. L., Tunney, M. M., Kahle, J. S., Waters, V. J., Elborn, J. S., Bell, S. C., Flume, P. A., et al. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.01.008 · Cited by 121
Effect of Azithromycin on Systemic Markers of Inflammation in Patients With Cystic Fibrosis Uninfected With Pseudomonas aeruginosa
Ratjen, F. A., Saiman, L. M., Mayer-Hamblett, N., Lands, L. C., Kloster, M., Thompson, V., Emmett, P., Marshall, B. C., Accurso, F. J., Sagel, S. D., et al. (2012) · CHEST Journal · doi:10.1378/chest.12-0628 · Cited by 120
Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis (PRESIS). A Randomized, Double-Blind, Controlled Study
Stahl, M., Wielpütz, M. O., Ricklefs, I., Dopfer, C., Barth, S., Schlegtendal, A., Graeber, S. Y., Sommerburg, O., Diekmann, G., Hüsing, J., et al. (2018) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201807-1203oc · Cited by 116
The Spectrum of CFTR Variants in Nonwhite Cystic Fibrosis Patients
Schrijver, I., Pique, L. M., Graham, S., Pearl, M., Cherry, A. M., & Kharrazi, M. (2015) · Journal of Molecular Diagnostics · doi:10.1016/j.jmoldx.2015.07.005 · Cited by 113
Good effect of IgY against Pseudomonas aeruginosa infections in cystic fibrosis patients
Nilsson, E., Larsson, A., Olesen, H. V., Wejåker, P., & Kollberg, H. (2008) · Pediatric Pulmonology · doi:10.1002/ppul.20875 · Cited by 111
Antifungal therapies for allergic bronchopulmonary aspergillosis in people with cystic fibrosis
Elphick, H., & Southern, K. W. (2016) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd002204.pub4 · Cited by 110
Pulmonary exacerbations in cystic fibrosis
Stenbit, A. E., & Flume, P. A. (2011) · Current Opinion in Pulmonary Medicine · doi:10.1097/mcp.0b013e32834b8c04 · Cited by 105
Dornase alfa for cystic fibrosis
Yang, C., & Montgomery, M. (2018) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd001127.pub4 · Cited by 101
Antipseudomonal Bacteriophage Reduces Infective Burden and Inflammatory Response in Murine Lung
Pabary, R., Singh, C., Morales, S., Bush, A., Alshafi, K., Bilton, D., Alton, E. W. F. W., Smithyman, A. M., & Davies, J. C. (2015) · Antimicrobial Agents and Chemotherapy · doi:10.1128/aac.01426-15 · Cited by 101
Self-citation
Azithromycin for Early Pseudomonas Infection in Cystic Fibrosis. The OPTIMIZE Randomized Trial
Mayer-Hamblett, N., Retsch‐Bogart, G. Z., Kloster, M., Accurso, F. J., Rosenfeld, M., Albers, G. M., Black, P., Brown, P. S., Cairns, A., Davis, S. D., et al. (2018) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201802-0215oc · Cited by 99
Airway surface liquid pH is not acidic in children with cystic fibrosis
Schultz, A., Puvvadi, R., Borisov, S. M., Shaw, N. C., Klimant, I., Berry, L. J., Montgomery, S., Nguyen, T., Kreda, S. M., Kicic, A., et al. (2017) · Nature Communications · doi:10.1038/s41467-017-00532-5 · Cited by 98
Cystic fibrosis related diabetes in Europe: prevalence, risk factors and outcome; Olesen et al
Olesen, H. V., Drevinek, P., Gulmans, V. A., Hatziagorou, E., Jung, A., Mei-Zahav, M., Stojnic, N., Thomas, M., & Zolin, A. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.10.009 · Cited by 95
Aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis, mild lung impairment, and P. aeruginosa
Wainwright, C. E., Quittner, A. L., Geller, D. E., Nakamura, C., Wooldridge, J. L., Gibson, R. L., Lewis, S., & Montgomery, A. B. (2011) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2011.02.007 · Cited by 94
Pulmonary Aspergillosis: An Evolving Challenge for Diagnosis and Treatment
Russo, A., Tiseo, G., Falcone, M., & Menichetti, F. (2020) · Infectious Diseases and Therapy · doi:10.1007/s40121-020-00315-4 · Cited by 93
A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR
Tosco, A., De Gregorio, F., Esposito, S., De Stefano, D., Sana, I., Ferrari, E., Sepe, A., Salvadori, L., Buonpensiero, P., Di Pasqua, A., et al. (2016) · Cell Death & Differentiation · doi:10.1038/cdd.2016.22 · Cited by 92
Inhaled anti-pseudomonal antibiotics for long-term therapy in cystic fibrosis
Smith, S., Rowbotham, N. J., & Regan, K. H. (2018) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd001021.pub3 · Cited by 92
Antibiotic Management of Lung Infections in Cystic Fibrosis. II. Nontuberculous Mycobacteria, Anaerobic Bacteria, and Fungi
Chmiel, J. F., Aksamit, T. R., Chotirmall, S. H., Dasenbrook, E. C., Elborn, J. S., LiPuma, J. J., Ranganathan, S., Waters, V. J., & Ratjen, F. A. (2014) · Annals of the American Thoracic Society · doi:10.1513/annalsats.201405-203as · Cited by 90
Standardized Treatment of Pulmonary Exacerbations (STOP) study: Observations at the initiation of intravenous antibiotics for cystic fibrosis pulmonary exacerbations
Sanders, D. B., Solomon, G. M., Beckett, V. V., West, N. E., Daines, C. L., Heltshe, S. L., VanDevanter, D. R., Spahr, J. E., Gibson, R. L., Nick, J. A., et al. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.04.005 · Cited by 87
Defining antimicrobial resistance in cystic fibrosis
Kidd, T. J., Cantón, R., Ekkelenkamp, M. B., Johansen, H. K., Gilligan, P. H., LiPuma, J. J., Bell, S. C., Elborn, J. S., Flume, P. A., VanDevanter, D. R., et al. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.08.014 · Cited by 86
A New Class of Safe Oligosaccharide Polymer Therapy To Modify the Mucus Barrier of Chronic Respiratory Disease
Pritchard, M., Powell, L. C., Menzies, G. E., Lewis, P. D., Hawkins, K. M., Wright, C. J., Doull, I. J. M., Walsh, T. R., Onsøyen, E., Dessen, A., et al. (2016) · Molecular Pharmaceutics · doi:10.1021/acs.molpharmaceut.5b00794 · Cited by 85
The Delta F508 Mutation in Cystic Fibrosis and Impact on Sinus Development
Woodworth, B. A., Ahn, C. N., Flume, P. A., & Schlosser, R. J. (2007) · American Journal of Rhinology · doi:10.2500/ajr.2007.21.2905 · Cited by 84
Positive expiratory pressure physiotherapy for airway clearance in people with cystic fibrosis
McIlwaine, M. P., Button, B., & Nevitt, S. J. (2019) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd003147.pub5 · Cited by 82
Neonates with cystic fibrosis have a reduced nasal liquid pH; A small pilot study
Alaiwa, M. H. A., Beer, A. M., Pezzulo, A. A., Launspach, J. L., Horan, R. A., Stoltz, D. A., Starner, T. D., Welsh, M. J., & Zabner, J. (2014) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2013.12.006 · Cited by 82
Airway surface liquid acidification initiates host defense abnormalities in Cystic Fibrosis
Simonin, J., Bille, E., Crambert, G., Noël, S., Dréano, E., Edwards, A., Hatton, A., Pranke, I. M., Villeret, B., Cottart, C., et al. (2019) · Scientific Reports · doi:10.1038/s41598-019-42751-4 · Cited by 82
The emerging burden of liver disease in cystic fibrosis patients: A UK nationwide study
Toledano, M. B., Mukherjee, S. K., Howell, J. A., Westaby, D., Khan, S. A., Bilton, D., & Simmonds, N. J. (2019) · PLoS ONE · doi:10.1371/journal.pone.0212779 · Cited by 81
Cysteamine re-establishes the clearance of Pseudomonas aeruginosa by macrophages bearing the cystic fibrosis-relevant F508del-CFTR mutation
Ferrari, E., Monzani, R., Villella, V. R., Esposito, S., Saluzzo, F., Rossin, F., D’Eletto, M., Tosco, A., Gregorio, F. D., Izzo, V., et al. (2017) · Cell Death and Disease · doi:10.1038/cddis.2016.476 · Cited by 77
ENaC inhibition in cystic fibrosis: potential role in the new era of CFTR modulator therapies
Mall, M. A. (2020) · European Respiratory Journal · doi:10.1183/13993003.00946-2020 · Cited by 77
Diagnosis of Cystic Fibrosis in Nonscreened Populations
Sosnay, P. R., White, T. B., Farrell, P. M., Ren, C. L., Derichs, N., Howenstine, M. S., Nick, J. A., & Boeck, K. D. (2017) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2016.09.068 · Cited by 76
Predictive factors for lumacaftor/ivacaftor clinical response
Masson, A., Schneider‐Futschik, E. K., Baatallah, N., Nguyen‐Khoa, T., Girodon, E., Hatton, A., Flament, T., Bourgeois, M. L., Chedevergne, F., Bailly-Botuha, C., et al. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.12.011 · Cited by 75
Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54
Rowe, S. M., Sloane, P. A., Tang, L. P., Backer, K., Mazur, M., Buckley-Lanier, J. A., Nudelman, I., Белахов, В. В., Bebők, Z., Schwiebert, E. M., et al. (2011) · Journal of Molecular Medicine · doi:10.1007/s00109-011-0787-6 · Cited by 74
A phase 3, multi-center, multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of levofloxacin inhalation solution (APT-1026) in stable cystic fibrosis patients
Flume, P. A., VanDevanter, D. R., Morgan, E. E., Dudley, M. N., Loutit, J., Bell, S. C., Kerem, E., Fischer, R., Smyth, A. R., Aaron, S. D., et al. (2016) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2015.12.004 · Cited by 74
The Physiology and Pathophysiology of Pancreatic Ductal Secretion
Pallagi, P., Hegyi, P. J., & Rakonczay, Z. (2015) · Pancreas · doi:10.1097/mpa.0000000000000421 · Cited by 74
The role for neutrophil extracellular traps in cystic fibrosis autoimmunity
Skopelja‐Gardner, S., Hamilton, B. J., Jones, J. D. G., Yang, M., Mamula, M. J., Ashare, A., Gifford, A. H., & Rigby, W. F. C. (2016) · JCI Insight · doi:10.1172/jci.insight.88912 · Cited by 74
Failure to conceive in women with CF is associated with pancreatic insufficiency and advancing age
Shteinberg, M., Lulu, A. B., Downey, D. G., Blumenfeld, Z., Rousset‐Jablonski, C., Perceval, M., Colombo, A. I. M., Stein, N., Livnat, G., Gur, M., et al. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.10.009 · Cited by 74
Self-citation
Growth and Nutrition in Cystic Fibrosis
Brownell, J. N., Bashaw, H., & Stallings, V. A. (2019) · Seminars in Respiratory and Critical Care Medicine · doi:10.1055/s-0039-1696726 · Cited by 72
Cystic fibrosis bone disease treatment: Current knowledge and future directions
Putman, M. S., Anabtawi, A., Le, T. N. H., Tangpricha, V., & Sermet‐Gaudelus, I. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.08.017 · Cited by 72
A Nanoscale Characterization of the Interaction of a Novel Alginate Oligomer with the Cell Surface and Motility of Pseudomonas aeruginosa
Powell, L. C., Pritchard, M., Emanuel, C., Onsøyen, E., Rye, P. D., Wright, C. J., Hill, K. E., & Thomas, D. W. (2013) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/rcmb.2013-0287oc · Cited by 68
Pooled analysis of two large randomised phase III inhaled mannitol studies in cystic fibrosis
Bilton, D., Bellon, G., Charlton, B., Cooper, P., Boeck, K. D., Flume, P. A., Fox, H. G., Gallagher, C. G., Geller, D. E., Haarman, E. G., et al. (2012) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2012.11.002 · Cited by 68
Pharmaceuticals Targeting Nonsense Mutations in Genetic Diseases
Rowe, S. M., & Clancy, J. P. (2009) · BioDrugs · doi:10.2165/00063030-200923030-00003 · Cited by 67
Is chest CT useful in newborn screened infants with cystic fibrosis at 1 year of age?
Thia, L. P., Calder, A. D., Stocks, J., Bush, A., Owens, C. M., Wallis, C., Young, C., Sullivan, Y., Wade, A. M., McEwan, A. I., et al. (2013) · Thorax · doi:10.1136/thoraxjnl-2013-204176 · Cited by 67
SharedPseudomonas aeruginosagenotypes are common in Australian cystic fibrosis centres
Kidd, T. J., Ramsay, K. A., Hu, H., Marks, G. B., Wainwright, C. E., Bye, P. T. P., Elkins, M. R., Robinson, P. J., Rose, B. R., Wilson, J. W., et al. (2012) · European Respiratory Journal · doi:10.1183/09031936.00060512 · Cited by 67
Integrating the multiple breath washout test into international multicentre trials
Saunders, C. J., Jensen, R., Robinson, P. D., Stanojevic, S., Klingel, M. L., Short, C., Davies, J. C., & Ratjen, F. A. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.11.006 · Cited by 65
Self-citation
The evolution of lung transplantation for cystic fibrosis: A 2017 update
Snell, G. I., Reed, A. K., Stern, M., & Hadjiliadis, D. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.06.008 · Cited by 64
Ajulemic acid: potential treatment for chronic inflammation
BURSTEIN, S. H. (2018) · Pharmacology Research & Perspectives · doi:10.1002/prp2.394 · Cited by 63
Pulmonary Outcomes Associated with Long-Term Azithromycin Therapy in Cystic Fibrosis
Nichols, D. P., Odem‐Davis, K., Cogen, J. D., Goss, C. H., Ren, C. L., Skalland, M., Somayaji, R., & Heltshe, S. L. (2019) · American Journal of Respiratory and Critical Care Medicine · doi:10.1164/rccm.201906-1206oc · Cited by 63
Characterization of Inpatient Cystic Fibrosis Pulmonary Exacerbations
Cogen, J. D., Oron, A. P., Gibson, R. L., Hoffman, L. R., Kronman, M. P., Ong, T., & Rosenfeld, M. (2017) · PEDIATRICS · doi:10.1542/peds.2016-2642 · Cited by 62
Induced sputum compared to bronchoalveolar lavage in young, non-expectorating cystic fibrosis children
Blau, H., Linnane, B. M., Carzino, R., Tannenbaum, E., Skoric, B., Robinson, P. J., ROBERTSON, C. F., & Ranganathan, S. (2013) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2013.05.013 · Cited by 61
Open label study of inhaled aztreonam for Pseudomonas eradication in children with cystic fibrosis: The ALPINE study
Tiddens, H. A. W. M., Boeck, K. D., Clancy, J., Fayon, M. J., Arets, H. G. M., Bresnik, M., Derchak, A., Lewis, S. A., & Oermann, C. M. (2014) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2014.06.003 · Cited by 61
Investigating transmission of Mycobacterium abscessus amongst children in an Australian cystic fibrosis centre
Yan, J., Kevat, A., Martínez, E., Teese, N., Johnson, K., Ranganathan, S., Harrison, J., Massie, J., & Daley, A. J. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.02.011 · Cited by 60
Cysteamine (Lynovex®), a novel mucoactive antimicrobial & antibiofilm agent for the treatment of cystic fibrosis
Charrier, C., Rodger, C., Robertson, J., Kowalczuk, A., Shand, N., Fraser-Pitt, D. J., Mercer, D. K., & O′Neil, D. A. (2014) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-014-0189-2 · Cited by 59
Inconclusive diagnosis after a positive newborn bloodspot screening result for cystic fibrosis; clarification of the harmonised international definition
Southern, K. W., Barben, J., Gärtner, S., Munck, À., Castellani, C., Mayell, S. J., Davies, J. C., Winters, V., Murphy, J. P., Salinas, D. B., et al. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.04.010 · Cited by 58
Self-citation
End of life care for patients with cystic fibrosis
Sands, D., Repetto, T., Dupont, L., Korzeniewska, A., Catastini, P., & Madge, S. L. (2011) · Journal of Cystic Fibrosis · doi:10.1016/s1569-1993(11)60007-6 · Cited by 57
Recent advances in developing therapeutics for cystic fibrosis
Strug, L. J., Stephenson, A. L., Panjwani, N., & Harris, A. (2018) · Human Molecular Genetics · doi:10.1093/hmg/ddy188 · Cited by 56
Amplifiers co-translationally enhance CFTR biosynthesis via PCBP1-mediated regulation of CFTR mRNA
Dukovski, D., Villella, A., Bastos, C., King, R. W., Finley, D. J., Kelly, J. W., Morimoto, R. I., Hartl, F. U., Muñoz, B., Lee, P., et al. (2020) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2020.02.006 · Cited by 54
Genetic medicines for CF: Hype versus reality
Alton, E. W. F. W., Boyd, A. C., Davies, J. C., Gill, D. R., Griesenbach, U., Harrison, P. T., Henig, N., Higgins, T., Hyde, S. C., Innes, J. A., et al. (2016) · Pediatric Pulmonology · doi:10.1002/ppul.23543 · Cited by 53
Self-citation
Intestinal organoids for Cystic Fibrosis research
de Poel, E., Lefferts, J. W., & Beekman, J. M. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.11.002 · Cited by 51
Quorum‐sensing inhibition abrogates the deleterious impact of Pseudomonas aeruginosa on airway epithelial repair
Ruffin, M., Bilodeau, C., Maillé, É., LaFayette, S. L., McKay, G. A., Trinh, N. T. N., Beaudoin, T., Desrosiers, M., Rousseau, S., Nguyen, D. M., et al. (2016) · The FASEB Journal · doi:10.1096/fj.201500166r · Cited by 50
Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis
Aslam, A. A., Higgins, C., Sinha, I. P., & Southern, K. W. (2017) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd012040.pub2 · Cited by 49
Deep resequencing of CFTR in 762 F508del homozygotes reveals clusters of non-coding variants associated with cystic fibrosis disease traits
Vecchio-Pagán, B., Blackman, S. M., Lee, M., Atalar, M., Pellicore, M. J., Pace, R. G., Franca, A., Raraigh, K. S., Sharma, N., Knowles, M. R., et al. (2016) · Human Genome Variation · doi:10.1038/hgv.2016.38 · Cited by 45
Energy balance and obesity in individuals with cystic fibrosis
Litvin, M., Yoon, J. C., Leey, J., Blackman, S. M., & Brennan, A. L. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.08.015 · Cited by 44
Nutritional Supplementation in Treatment of Cystic Fibrosis of the Pancreas
Allan, J. D. (1973) · Archives of Pediatrics and Adolescent Medicine · doi:10.1001/archpedi.1973.02110190018004 · Cited by 44
Phase I Studies of Acebilustat: Biomarker Response and Safety in Patients with Cystic Fibrosis
Elborn, J. S., Horsley, A. R., MacGregor, G., Bilton, D., Grosswald, R., Ahuja, S., & Springman, E. B. (2016) · Clinical and Translational Science · doi:10.1111/cts.12428 · Cited by 44
Bone involvement in young adults with cystic fibrosis awaiting lung transplantation for end-stage respiratory failure
Cairoli, E., Eller-Vainicher, C., Morlacchi, L. C., Tarsia, P., Rossetti, V., Pappalettera, M., Arosio, M., Chiodini, I., & Blasi, F. (2019) · Osteoporosis International · doi:10.1007/s00198-019-04893-z · Cited by 43
Underweight Patients With Cystic Fibrosis Have Acceptable Survival Following Lung Transplantation
Ramos, K. J., Kapnadak, S. G., Bradford, M. C., Somayaji, R., Morrell, E. D., Pilewski, J. M., Lease, E. D., Mulligan, M. S., Aitken, M. L., Gries, C. J., et al. (2020) · Chest · doi:10.1016/j.chest.2019.11.043 · Cited by 42
Modifier genes in cystic fibrosis-related liver disease
Debray, D., Corvol, H., & Housset, C. (2018) · Current Opinion in Gastroenterology · doi:10.1097/mog.0000000000000508 · Cited by 42
Fenretinide differentially modulates the levels of long- and very long-chain ceramides by downregulating Cers5 enzyme: evidence from bench to bedside
Garić, D., Sanctis, J. B. D., Wojewodka, G., Houle, D., Cupri, S., Abu‐Arish, A., Hanrahan, J. W., Hajdúch, M., Matouk, E., & Radzioch, D. (2017) · Journal of Molecular Medicine · doi:10.1007/s00109-017-1564-y · Cited by 41
The influence of CFTR complex alleles on precision therapy of cystic fibrosis
Chevalier, B., & Hinzpeter, A. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.12.008 · Cited by 41
Nutritional status, nutrient intake and use of enzyme supplements in paediatric patients with Cystic Fibrosis; a European multicentre study with reference to current guidelines
Calvo‐Lerma, J., Hulst, J. M., Asseiceira, I., Claes, I., Garriga, M. J., Colombo, C., Fornés, V., Woodcock, S., Martins, T. F., Boon, M., et al. (2017) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.03.005 · Cited by 40
Standardized Measurement of Nasal Membrane Transepithelial Potential Difference (NPD)
Solomon, G. M., Bronsveld, I., Hayes, K., Wilschanski, M. A., Melotti, P., Rowe, S. M., & Sermet‐Gaudelus, I. (2018) · Journal of Visualized Experiments · doi:10.3791/57006 · Cited by 40
Omalizumab for asthma and allergic bronchopulmonary aspergillosis in adults with cystic fibrosis
Koutsokera, A., Corriveau, S., Sykes, J. R., Coriati, A., Cortes, D., Vadas, P., Chaparro, C., McIntyre, K., Tullis, E. D., & Stephenson, A. L. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.07.011 · Cited by 38
Prevalence and clinical significance of auto-antibodies in adults with cystic fibrosis
Lachenal, F., Nkana, K. N. M., NOVE-JOSSERAND, R., Fabien, N., & Durieu, I. (2009) · European Respiratory Journal · doi:10.1183/09031936.00006009 · Cited by 38
Beyond pancreatic insufficiency and liver disease in cystic fibrosis
Demeyer, S., Boeck, K. D., Witters, P., & Cosaert, K. (2016) · European Journal of Pediatrics · doi:10.1007/s00431-016-2719-5 · Cited by 37
Implementing the international committee on mental health in cystic fibrosis (ICMH) guidelines: screening accuracy and referral-treatment pathways
Verkleij, M., de Winter, D., Hurley, M. A., & Abbott, J. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.02.005 · Cited by 37
Acid Stimulation Reduces Bactericidal Activity of Surface Liquid in Cultured Human Airway Epithelial Cells
Nakayama, K., Jia, Y. X., Hirai, H., Shinkawa, M., Yamaya, M., Sekizawa, K., & Sasaki, H. (2002) · American Journal of Respiratory Cell and Molecular Biology · doi:10.1165/ajrcmb.26.1.4425 · Cited by 37
Young patients with cystic fibrosis demonstrate subtle alterations of the cardiovascular system
Eising, J. B., van der Ent, C. K., Teske, A. J., Vanderschuren, M. M., Uiterwaal, C. S. P. M., & Meijboom, F. J. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2017.12.009 · Cited by 37
Probiotics in cystic fibrosis patients: A double blind crossover placebo controlled study
Biervliet, S. V., Hauser, B., Verhulst, S. L., Stepman, H. C. M., Delanghe, J., Warzée, J.-P., Pot, B., de Wiele, T. V., & Wilschanski, M. A. (2018) · Clinical Nutrition ESPEN · doi:10.1016/j.clnesp.2018.06.008 · Cited by 36
Liprotamase Long‐term Safety and Support of Nutritional Status in Pancreatic‐insufficient Cystic Fibrosis
Borowitz, D., Stevens, C. G., Brettman, L. R., Campion, M., Wilschanski, M. A., Thompson, H. C., & Liprotamase 767 Study Group (2011) · Journal of Pediatric Gastroenterology and Nutrition · doi:10.1097/mpg.0b013e31823315d1 · Cited by 30
Upper versus lower airway microbiome and metagenome in children with cystic fibrosis and their correlation with lung inflammation
Kirst, M. E., Baker, D., Li, E., Abu‐Hasan, M., & Wang, G. P. (2019) · PLoS ONE · doi:10.1371/journal.pone.0222323 · Cited by 29
ISPAD Clinical Practice Consensus Guidelines 2018: What is new in diabetes care?
Ethel Codner, D., Acerini, C. L., Craig, M. E., Hofer, S. E., & Maahs, D. M. (2018) · Pediatric Diabetes · doi:10.1111/pedi.12759 · Cited by 28
Association Between Fat‐Free Mass and Pulmonary Function in Patients With Cystic Fibrosis: A Narrative Review
Gomes, A., Hutcheon, D. A., & Ziegler, J. (2019) · Nutrition in Clinical Practice · doi:10.1002/ncp.10251 · Cited by 27
Predictors of pulmonary exacerbation treatment in cystic fibrosis
Sanders, D. B., Ostrenga, J. S., Rosenfeld, M., Fink, A. K., Schechter, M. S., Sawicki, G. S., Flume, P. A., & Morgan, W. J. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.06.008 · Cited by 26
Newborn screening programmes for cystic fibrosis
Southern, K. W., & Littlewood, J. M. (2003) · Paediatric Respiratory Reviews · doi:10.1016/s1526-0542(03)00093-9 · Cited by 25
Safety and Efficacy of a Novel Microbial Lipase in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis: A Randomized Controlled Clinical Trial
Heubi, J. E., Schaeffer, D., Ahrens, R. C., Sollo, N., Strausbaugh, S. D., Graff, G. R., Jain, R., Witte, S., & Forssmann, K. (2016) · The Journal of Pediatrics · doi:10.1016/j.jpeds.2016.05.049 · Cited by 25
Outcomes in cystic fibrosis lung transplant recipients infected with organisms labeled as pan-resistant: An ISHLT Registry‒based analysis
Lay, C., Law, N., Holm, A. M., Benden, C., & Aslam, S. (2019) · The Journal of Heart and Lung Transplantation · doi:10.1016/j.healun.2019.01.1306 · Cited by 24
Sildenafil improves vascular endothelial function in patients with cystic fibrosis
Rodriguez‐Miguelez, P., Lee, N., Tucker, M. A., Csányi, G., McKie, K. T., Forseen, C. J., & Harris, R. A. (2018) · American Journal of Physiology-Heart and Circulatory Physiology · doi:10.1152/ajpheart.00301.2018 · Cited by 24
Palliative Care Needs of Individuals with Cystic Fibrosis: Perspectives of Multiple Stakeholders
Dellon, E. P., Basile, M. J., Hobler, M. R., Georgiopoulos, A. M., Chen, E. Y., Goggin, J. L., Goss, C. H., Hempstead, S. E., Faro, A., & Kavalieratos, D. (2020) · Journal of Palliative Medicine · doi:10.1089/jpm.2019.0464 · Cited by 22
How to use: bacterial cultures in diagnosing lower respiratory tract infections in cystic fibrosis
Ahmed, B., Bush, A., & Davies, J. C. (2013) · Archives of Disease in Childhood Education & Practice · doi:10.1136/archdischild-2012-303408 · Cited by 21
Self-citation
Dissociation of systemic and mucosal autoimmunity in cystic fibrosis
Theprungsirikul, J., Skopelja‐Gardner, S., Meagher, R. E., Clancy, J., Zemanick, E. T., Ashare, A., & Rigby, W. F. C. (2019) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2019.06.006 · Cited by 20
Ten years of chest MRI for patients with cystic fibrosis
Leutz-Schmidt, P., Eichinger, M., Stahl, M., Sommerburg, O., Biederer, J., Kauczor, H., Puderbach, M., Mall, M. A., & Wielpütz, M. O. (2019) · Der Radiologe · doi:10.1007/s00117-019-0553-2 · Cited by 20
Short-Term Effects of Chest Physiotherapy on Pulmonary Function in Children with Cystic Fibrosis
Weller, P. H., Bush, E., Preece, M. A., & Matthew, D. J. (2009) · Respiration · doi:10.1159/000194251 · Cited by 19
Analysis of a large cohort of cystic fibrosis patients with severe liver disease indicates lung function decline does not significantly differ from that of the general cystic fibrosis population
Polineni, D., Piccorelli, A. V., Hannah, W. B., Dalrymple, S. N., Pace, R. G., Durie, P. R., Ling, S. C., Knowles, M. R., & Stonebraker, J. R. (2018) · PLoS ONE · doi:10.1371/journal.pone.0205257 · Cited by 18
Self-citation
Adults with cystic fibrosis have deficits in bone structure and strength at the distal tibia despite similar size and measuring standard and relative sites
Nishiyama, K. K., Agarwal, S., Kepley, A. L., Rosete, F., Hu, Y., Guo, X., Keating, C. L., DiMango, E., & Shane, E. J. (2017) · Bone · doi:10.1016/j.bone.2017.11.006 · Cited by 17
Effects of a primary palliative care intervention on quality of life and mental health in cystic fibrosis
Friedman, D., Linnemann, R. W., Altstein, L. L., Georgiopoulos, A. M., Islam, S., Bach, K., John, A. S., Fracchia, M. S., Neuringer, I. P., Lapey, A., et al. (2019) · Pediatric Pulmonology · doi:10.1002/ppul.24311 · Cited by 17
Clinical manifestations and risk factors of arthropathy in cystic fibrosis
Roehmel, J. F., Kallinich, T., Staab, D., & Schwarz, C. (2019) · Respiratory Medicine · doi:10.1016/j.rmed.2019.01.003 · Cited by 17
Infection prevention and control in cystic fibrosis: a systematic review of interventions
Rowbotham, N. J., Palser, S. C., Smith, S. J., & Smyth, A. R. (2019) · Expert Review of Respiratory Medicine · doi:10.1080/17476348.2019.1595594 · Cited by 17
Elastic aortic properties in cystic fibrosis adults without cardiovascular risk factors: A case‐control study
Vizzardi, E., Sciatti, E., Bonadei, I., Menotti, E., Prati, F., Scodro, M., Dallapellegrina, L., Berlendis, M., Poli, P., Padoan, R., et al. (2019) · Echocardiography · doi:10.1111/echo.14375 · Cited by 16
Clinical outcomes for cystic fibrosis patients with Pseudomonas aeruginosa cross‐infections
Milczewska, J., Wołkowicz, T., Zacharczuk, K., Mierzejewska, E., Kwiatkowska, M., Walicka‐Serzysko, K., & Sands, D. (2019) · Pediatric Pulmonology · doi:10.1002/ppul.24535 · Cited by 16
Prevalence of Bactericidal/Permeability-Increasing Protein Autoantibodies in Cystic Fibrosis Patients: Systematic Review and Meta-Analysis
IWUJI, K. C., Larumbe‐Zabala, E., Bijlani, S., NUGENT, K. M., Kanu, A., Manning, E., & Solis, X. (2019) · Pediatric Allergy Immunology and Pulmonology · doi:10.1089/ped.2018.0970 · Cited by 16
Mental Health Screening of Medically-Admitted Patients With Cystic Fibrosis
García, G., Snell, C., Sawicki, G. S., & Simons, L. E. (2017) · Psychosomatics · doi:10.1016/j.psym.2017.08.010 · Cited by 15
Morphometry versus densitometry ? a comparison by use of casts of human coronary arteries
Doriot, P. A., Suilen, C., Guggenheim, N., Dorsaz, P. ‐., Chappuis, F., & Rutishauser, W. (1992) · International journal of cardiac imaging · doi:10.1007/bf01137533 · Cited by 14
The effect of enteral tube feeding in cystic fibrosis: A registry based study
Libeert, D., Declercq, D., Wanyama, S. S., Thomas, M., daele, S. V., Baets, F. D., & Biervliet, S. V. (2018) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2018.01.004 · Cited by 14
Challenging scenarios in nontuberculous mycobacterial infection in cystic fibrosis
Martiniano, S. L., Esther, C. R., HAWORTH, C. S., Kasperbauer, S. H., Zemanick, E. T., & Caverly, L. J. (2019) · Pediatric Pulmonology · doi:10.1002/ppul.24604 · Cited by 13
Vitamin K supplementation for cystic fibrosis
Jagannath, V. A., Thaker, V., Chang, A. B., & Price, A. I. (2020) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd008482.pub6 · Cited by 12
Highlights from the 2016 North American Cystic Fibrosis Conference
Zemanick, E. T., Daines, C. L., Dellon, E. P., Esther, C. R., Kinghorn, B., Ong, T., & Muhlebach, M. S. (2017) · Pediatric Pulmonology · doi:10.1002/ppul.23707 · Cited by 11
Burkholderia cepacia complex and limited cutaneous vasculitis in patients with cystic fibrosis: a case series
Kayria, M., Chris, O., Dhasmana, D., Morar, N., Hodson, M. E., Gyi, K. M., Bilton, D., & Simmonds, N. J. (2017) · JRSM Open · doi:10.1177/2054270417692732 · Cited by 10
An innovative phase II trial to establish proof of efficacy and optimal dose of a new inhaled epithelial sodium channel inhibitor BI 1265162 in adults and adolescents with cystic fibrosis: BALANCE-CFTM1
Goss, C. H., Jain, R., Seibold, W., Picard, A.-C., Hsu, M.-C., Gupta, A., & Fajac, I. (2020) · ERJ Open Research · doi:10.1183/23120541.00395-2020 · Cited by 9
Cystic Fibrosis from Childhood to Adulthood
Hota, P. V., & Madan, R. (2020) · Radiologic Clinics of North America · doi:10.1016/j.rcl.2019.12.003 · Cited by 8
Five Cases of Cystic Fibrosis of the Pancreas
Wilmers, M. J., Mackay, H., & ANDERSON, I. M. (1950) · Proceedings of the Royal Society of Medicine · doi:10.1177/003591575004301114 · Cited by 4
Salt Excretion in Sweat in Cystic Fibrosis
Siegenthaler, P., Dubach, U. C., & Haller, R. D. (1963) · JAMA · doi:10.1001/jama.1963.03710130074023 · Cited by 3
Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis
Aslam, A., Sinha, I. P., & Southern, K. W. (2016) · Cochrane Database of Systematic Reviews · doi:10.1002/14651858.cd012040

Research works printed but not linked: 2

Saunders, 2016, Developments in multibreath washout testing in children with cystic fibrosis, Curr Med Res Opin, 33, 1
2015, Symposium Summaries, Pediatr Pulmonol, 50, S108

Sorted by citations

CRISPR/Cas9 therapeutics: progress and prospects
Li, T., Yang, Y., Qi, H., Cui, W., Zhang, L., Fu, X., He, X., Liu, M., Li, P., & Yu, T. (2023) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-023-01309-7 · Cited by 824
Worldwide rates of diagnosis and effective treatment for cystic fibrosis
Guo, J., Garratt, A., & Hill, A. M. (2022) · Journal of Cystic Fibrosis · doi:10.1016/j.jcf.2022.01.009 · Cited by 395
Physiology and pathophysiology of human airway mucus
Hill, D. B., Button, B. M., Rubinstein, M., & Boucher, R. C. (2022) · Physiological Reviews · doi:10.1152/physrev.00004.2021 · Cited by 406
Cystic Fibrosis
Ong, T., & Ramsey, B. W. (2023) · JAMA · doi:10.1001/jama.2023.8120 · Cited by 346
mRNA vaccines in disease prevention and treatment
Zhang, G., Tang, T., Chen, Y., Huang, X., & Liang, T. (2023) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-023-01579-1 · Cited by 273

See all 685

Authors: Shteinberg, M., Haq, I. J., Polineni, D., & Davies, J. C.
Affiliations: Technion – Israel Institute of Technology (IL)
Newcastle upon Tyne Hospitals NHS Foundation Trust (GB)
University of Kansas Medical Center (US)
Guy's and St Thomas' NHS Foundation Trust (GB)
ORCID: Shteinberg, Michal
Haq, Iram J
Polineni, Deepika
Davies, Jane C
Venue: The Lancet
Publisher: Elsevier BV
Published: 2021-06
Volume: 397
Issue: 10290
Pages: 2195-2211
ISSN: 0099-5355 (ISSN-L)
0140-6736
DOI: 10.1016/s0140-6736(20)32542-3
OpenAlex: W4205612199
Cited by: 682
References: 195
Citations by year: 2026: 96 · 2025: 132 · 2024: 147 · 2023: 160 · 2022: 131 · 2021: 14
FWCI: 18.21
Type: journal article
Language: en
Open access: green
doi.org
Licence: www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
Locations: The Lancet (publishedVersion)
PubMed (publishedVersion)
Newcastle University ePrints (Newcastle Univesity)
Funders: National Institute for Health and Care Research (NIHR201402, CL-2018-01-002)
Cystic Fibrosis Trust (SRC 014)
Academy of Medical Sciences (SGL024\1027)
Topics: Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Cystic Fibrosis Research Advances
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic and Kidney Cyst Diseases
Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Neonatal Respiratory Health Research
Keywords: cystic fibrosis, CFTR mutations, CFTR function, mucus hydration, chronic lung infection, exocrine pancreatic insufficiency, male infertility, cystic fibrosis-related diabetes, cystic fibrosis liver disease, newborn screening, sweat test, CFTR modulator therapy, mucociliary clearance, disease pathophysiology, multidisciplinary care
MeSH terms: Bicarbonates, Chlorides, Cystic Fibrosis, Humans, Life Expectancy, Mucociliary Clearance, Mutation, Exocrine Pancreatic Insufficiency, Genetic Therapy, Cystic Fibrosis Transmembrane Conductance Regulator, Disease Management

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

Cystic fibrosis

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Citations

Cites

Cited by

Related

Metadata

Record

About these checks