Re-imagining cystic fibrosis care: next generation thinking.

The European Respiratory Journal
Catherine RangTom Kotsimbos

Abstract

Cystic fibrosis (CF) is a common multi-system genetically inherited condition, predominately found in individuals of Caucasian decent. Since the identification of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gene in 1989, and the subsequent improvement in understanding of CF pathophysiology, significant increases in life-expectancy have followed. Initially this was related to improvements in the management and systems of care for treating the various affected organ systems. These cornerstone treatments are still essential for CF patients born today. However, over the last decade, the major advance has been in therapies that target the resultant genetic defect: the dysfunctional CFTR protein. Small molecule agents that target this dysfunctional protein via a variety of mechanisms have led to lung function improvements, reductions in pulmonary exacerbation rates and increases in weight and quality-of-life indices. As more patients receive these agents earlier and earlier in life, it is likely that general CF care will increasingly pivot around these specific therapies, although it is also likely that effects other than those identified in the initial trials will be discovered and need to be managed. Despite...Continue Reading

References

Apr 30, 1992·The New England Journal of Medicine·E KeremH Levison
Dec 17, 1992·The New England Journal of Medicine·P A NixonC F Doershuk
Jan 15, 1995·The Journal of Physiology·A W CuthbertM J Evans
Aug 19, 1999·Thorax·U GriesenbachE W Alton
Apr 15, 2000·Nature·P G Kopelman
Oct 12, 2000·Anais Da Academia Brasileira De Ciências·M M MoralesA G Lopes
Sep 14, 2001·Chest·T R Flotte, B L Laube
Oct 10, 2003·The New England Journal of Medicine·Michael WilschanskiEitan Kerem
Apr 2, 2004·Nephron. Clinical Practice·Glen P WestallJohn W Wilson
Oct 7, 2004·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Ludger DorlöchterGjermund Fluge
Nov 2, 2004·The European Respiratory Journal·K W Southern, P M Barker
Jan 11, 2005·The European Respiratory Journal·S J KingJ W Wilson
Sep 27, 2005·The Journal of Pediatrics·Dagmar Kastner-ColeSomnath Mukhopadhyay
Jan 20, 2006·The New England Journal of Medicine·Mark R ElkinsUNKNOWN National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group
Jan 31, 2006·American Journal of Physiology. Lung Cellular and Molecular Physiology·Fredrick Van GoorPaul Negulescu
Jul 15, 2006·American Journal of Respiratory and Critical Care Medicine·Michael WilschanskiPeter Durie
Nov 21, 2007·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·F P EdenboroughUNKNOWN European Cystic Fibrosis Society
Feb 29, 2008·Proceedings of the National Academy of Sciences of the United States of America·Adrian W R SerohijosJohn R Riordan
Jul 29, 2008·American Journal of Respiratory and Critical Care Medicine·Karen S McCoyA Bruce Montgomery
May 1, 2009·Human Genomics·Vasilis VasiliouDaniel W Nebert
Aug 6, 2009·The Journal of Histochemistry and Cytochemistry : Official Journal of the Histochemistry Society·Yong GuoJiang Gu
Oct 23, 2009·Proceedings of the National Academy of Sciences of the United States of America·Fredrick Van GoorPaul Negulescu
Feb 12, 2010·International Journal of Endocrinology·William B HallRobert M Aris
Jul 14, 2010·American Journal of Respiratory and Critical Care Medicine·Isabelle Sermet-GaudelusLangdon L Miller
Aug 27, 2010·Current Opinion in Pulmonary Medicine·Charles S Haworth
Sep 11, 2010·Current Opinion in Pulmonary Medicine·Peter A Sloane, Steven M Rowe
Dec 24, 2010·Annals of the New York Academy of Sciences·Garry R Cutting
Mar 1, 2011·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·A Geborek, L Hjelte
Mar 15, 2011·Oncotarget·Bart ThaciMaciej S Lesniak
Jun 17, 2011·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Isabelle Sermet-GaudelusCharles S Haworth
Oct 7, 2011·Proceedings of the National Academy of Sciences of the United States of America·Fredrick Van GoorPaul A Negulescu

❮ Previous
Next ❯

Citations

May 11, 2021·Frontiers in Immunology·Rafael I JaénPatricia Prieto
May 26, 2021·Pediatric Pulmonology·Nicholas J Antos, Adrienne P Savant
Jul 25, 2021·International Journal of Molecular Sciences·Kiera H HarwoodElena K Schneider-Futschik
Aug 20, 2021·European Journal of Medical Genetics·Connor D DaviesonClaire L Shovlin

❮ Previous
Next ❯

Related Concepts

Related Feeds

CFTR Mutant Structural Therapy

Over 1700 different mutations in the CFTR genes have been shown to cause cystic fibrosis. Here is the latest research on structural therapy for CFTR mutants.

© 2021 Meta ULC. All rights reserved