This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features!

Skip to main page content

Add to Collections

Your saved search

Name of saved search:

Search terms:

Test search terms

Would you like email updates of new search results?

Yes
No

Email: (change)

Frequency:

Which day?

Which day?

Report format:

Send at most:

Send even when there aren't any new results

Optional text in email:

Your RSS Feed

. 2017 Apr 15;195(8):979-984.

doi: 10.1164/rccm.201606-1250PP.

Cystic Fibrosis: The Dawn of a New Therapeutic Era

Sonya L Heltshe^{1

2}, Jonathan Cogen¹, Kathleen J Ramos³, Christopher H Goss^{1

3

2}

Affiliations

Affiliations

¹ 1 Division of Pediatric Pulmonology, Department of Pediatrics, and.
² 2 Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington.
³ 3 Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of Washington, Seattle, Washington; and.

PMID: 27710011
PMCID: PMC5422649
DOI: 10.1164/rccm.201606-1250PP

Cystic Fibrosis: The Dawn of a New Therapeutic Era

Sonya L Heltshe et al. Am J Respir Crit Care Med. 2017.

. 2017 Apr 15;195(8):979-984.

doi: 10.1164/rccm.201606-1250PP.

Authors

Sonya L Heltshe^{1

2}, Jonathan Cogen¹, Kathleen J Ramos³, Christopher H Goss^{1

3

2}

Affiliations

¹ 1 Division of Pediatric Pulmonology, Department of Pediatrics, and.
² 2 Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington.
³ 3 Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of Washington, Seattle, Washington; and.

PMID: 27710011
PMCID: PMC5422649
DOI: 10.1164/rccm.201606-1250PP

No abstract available

PubMed Disclaimer

Figures

Figure 1. — Figure 1.
Classes of cystic fibrosis transmembrane conductance regulator protein (CFTR) mutations. Note that class VI is not included in this figure. Adapted by permission from http://www.umd.be/CFTR/W_CFTR/gene.html with initial adaptation from Welsh and Smith (26) and Scriver and colleagues (63). Reprinted from Reference , by permission from the Cystic Fibrosis Foundation.

See this image and copyright information in PMC

Similar articles

Treating Specific Variants Causing Cystic Fibrosis.
Cutting GR. Cutting GR. JAMA. 2017 Dec 5;318(21):2130-2131. doi: 10.1001/jama.2017.16823. JAMA. 2017. PMID: 29209709 No abstract available.
Clinical trial research in focus: ensuring new cystic fibrosis drugs fulfil their potential.
Elborn JS, Davies J. Elborn JS, et al. Lancet Respir Med. 2017 Sep;5(9):681-683. doi: 10.1016/S2213-2600(17)30311-9. Lancet Respir Med. 2017. PMID: 28853396 No abstract available.
Breathing easier with combinations.
Azvolinsky A. Azvolinsky A. Nat Biotechnol. 2015 Nov;33(11):1128-30. doi: 10.1038/nbt.3397. Nat Biotechnol. 2015. PMID: 26544137 No abstract available.
Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
Schneider EK, Reyes-Ortega F, Li J, Velkov T. Schneider EK, et al. Clin Pharmacol Ther. 2017 Jan;101(1):130-141. doi: 10.1002/cpt.548. Epub 2016 Nov 23. Clin Pharmacol Ther. 2017. PMID: 27804127 Free PMC article. Review.
New Therapeutic Approaches to Modulate and Correct Cystic Fibrosis Transmembrane Conductance Regulator.
Ong T, Ramsey BW. Ong T, et al. Pediatr Clin North Am. 2016 Aug;63(4):751-64. doi: 10.1016/j.pcl.2016.04.006. Pediatr Clin North Am. 2016. PMID: 27469186 Free PMC article. Review.

See all similar articles

Cited by

Critical illness among adults with cystic fibrosis in Texas, 2004-2013: Patterns of ICU utilization, characteristics, and outcomes.
Oud L. Oud L. PLoS One. 2017 Oct 24;12(10):e0186770. doi: 10.1371/journal.pone.0186770. eCollection 2017. PLoS One. 2017. PMID: 29065161 Free PMC article.
Building a better biofilm - Formation of in vivo-like biofilm structures by Pseudomonas aeruginosa in a porcine model of cystic fibrosis lung infection.
Harrington NE, Sweeney E, Harrison F. Harrington NE, et al. Biofilm. 2020 Dec;2:100024. doi: 10.1016/j.bioflm.2020.100024. Biofilm. 2020. PMID: 33381751 Free PMC article.
Elastase Exocytosis by Airway Neutrophils Is Associated with Early Lung Damage in Children with Cystic Fibrosis.
Margaroli C, Garratt LW, Horati H, Dittrich AS, Rosenow T, Montgomery ST, Frey DL, Brown MR, Schultz C, Guglani L, Kicic A, Peng L, Scholte BJ, Mall MA, Janssens HM, Stick SM, Tirouvanziam R. Margaroli C, et al. Am J Respir Crit Care Med. 2019 Apr 1;199(7):873-881. doi: 10.1164/rccm.201803-0442OC. Am J Respir Crit Care Med. 2019. PMID: 30281324 Free PMC article.
Folding and Misfolding of Human Membrane Proteins in Health and Disease: From Single Molecules to Cellular Proteostasis.
Marinko JT, Huang H, Penn WD, Capra JA, Schlebach JP, Sanders CR. Marinko JT, et al. Chem Rev. 2019 May 8;119(9):5537-5606. doi: 10.1021/acs.chemrev.8b00532. Epub 2019 Jan 4. Chem Rev. 2019. PMID: 30608666 Free PMC article.
Organic Acids and Their Salts Potentiate the Activity of Selected Antibiotics against Pseudomonas aeruginosa Biofilms Grown in a Synthetic Cystic Fibrosis Sputum Medium.
Bao X, Bové M, Coenye T. Bao X, et al. Antimicrob Agents Chemother. 2022 Jan 18;66(1):e0187521. doi: 10.1128/AAC.01875-21. Epub 2021 Nov 22. Antimicrob Agents Chemother. 2022. PMID: 34807756 Free PMC article.

See all "Cited by" articles

References

1. Hoegger MJ, Fischer AJ, McMenimen JD, Ostedgaard LS, Tucker AJ, Awadalla MA, Moninger TO, Michalski AS, Hoffman EA, Zabner J, et al. Impaired mucus detachment disrupts mucociliary transport in a piglet model of cystic fibrosis. Science. 2014;345:818–822. - PMC - PubMed
1. Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Dřevínek P, Griese M, McKone EF, Wainwright CE, Konstan MW, et al. VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011;365:1663–1672. - PMC - PubMed
1. Wainwright CE, Elborn JS, Ramsey BW. Lumacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med. 2015;373:1783–1784. - PubMed
1. Elborn JS, Bell SC, Madge SL, Burgel PR, Castellani C, Conway S, De Rijcke K, Dembski B, Drevinek P, Heijerman HG, et al. Report of the European Respiratory Society/European Cystic Fibrosis Society task force on the care of adults with cystic fibrosis. Eur Respir J. 2016;47:420–428. - PubMed
1. MacKenzie T, Gifford AH, Sabadosa KA, Quinton HB, Knapp EA, Goss CH, Marshall BC. Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the Cystic Fibrosis Foundation Patient Registry. Ann Intern Med. 2014;161:233–241. - PMC - PubMed

Publication types

Actions
Actions
Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Substances

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Grants and funding

LinkOut - more resources

Full Text Sources
Other Literature Sources
- scite Smart Citations
Medical
- Genetic Alliance
- MedlinePlus Health Information