Therapeutic advances in cystic fibrosis in 2014

Revue de pneumologie clinique
S DuruptP Reix

Abstract

Twenty-five years after the cystic fibrosis (CF) gene identification, this discovery actually begins to benefit to patients. Increasing our knowledge on CFTR biology, as well as technical progress made in order to screen for new drugs have made therapeutic strategies move an important step forward. It is likely that in the forthcoming years, the panel of molecules available for CF patients will be larger, with new activators and potentiators. The disease by itself may consequently change in its natural history. CF is an example of the so-called personalized medicine, aiming to fit treatment according to patient's genetic background. Ongoing clinical trials may enlarge the actually limited eligible number of CF patients for new drugs such as ivacaftor. Beyond this exciting and promising new therapeutic approach, one may not push symptomatic treatments on the side. Improvements have been made for inhaled antibiotics administration, aiming to simplify patient's life; clinical trials using new molecules able to liquefy mucus or with anti-inflammatory properties are actually underway. One important next step in the care for CF will be to design and conduct early intervention trials in CF infants. Newborn screening program have been ...Continue Reading

References

Feb 15, 2008·Proceedings of the National Academy of Sciences of the United States of America·Ming DuDavid M Bedwell
Feb 23, 2008·La Revue de médecine interne·I Durieu, R Nove Josserand
May 6, 2008·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·C CastellaniJ S Elborn
Jul 29, 2008·American Journal of Respiratory and Critical Care Medicine·Karen S McCoyA Bruce Montgomery
Sep 13, 2008·American Journal of Respiratory and Critical Care Medicine·Barry M LinnaneUNKNOWN AREST-CF
Apr 18, 2009·American Journal of Respiratory and Critical Care Medicine·Peter D SlyUNKNOWN Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST-CF)
Oct 7, 2009·Expert Review of Anti-infective Therapy·Stephen KirkbyKaren McCoy
Oct 23, 2009·Proceedings of the National Academy of Sciences of the United States of America·Fredrick Van GoorPaul Negulescu
Jul 14, 2010·American Journal of Respiratory and Critical Care Medicine·Isabelle Sermet-GaudelusLangdon L Miller
Oct 22, 2010·Pediatric Pulmonology·Michael W KonstanGerhild Angyalosi
Nov 16, 2010·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Michael W KonstanDavid E Geller
Nov 19, 2010·The New England Journal of Medicine·Frank J AccursoBonnie W Ramsey
Apr 16, 2011·American Journal of Respiratory and Critical Care Medicine·Naveen PillarisettiUNKNOWN Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF)
Nov 4, 2011·The New England Journal of Medicine·Bonnie W RamseyUNKNOWN VX08-770-102 Study Group
Feb 2, 2012·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Haihui YuFredrick Van Goor
Mar 3, 2012·Chest·Patrick A FlumeUNKNOWN VX 08-770-104 Study Group
Apr 7, 2012·Nature Medicine·Taylor Sitarik Cohen, Alice Prince
May 1, 2012·Nature Reviews. Drug Discovery·Pamela B DavisPeter Kirkpatrick
May 23, 2012·JAMA : the Journal of the American Medical Association·Margaret RosenfeldUNKNOWN ISIS Study Group
Jun 16, 2012·Clinical Biochemistry·Bob LubambaTeresinha Leal
Jun 23, 2012·Frontiers in Pharmacology·Michael Wilschanski
Aug 14, 2012·BMC Medicine·Patrick A Flume, Donald R Van Devanter
Sep 11, 2012·Thorax·Steven M RoweBonnie W Ramsey
Dec 14, 2012·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·Diana BiltonUNKNOWN CF301 and CF302 Investigators
Jan 2, 2013·Chest·Lucas R Hoffman, Bonnie W Ramsey
Mar 5, 2013·European Respiratory Review : an Official Journal of the European Respiratory Society·Nico Derichs
Mar 5, 2013·European Respiratory Review : an Official Journal of the European Respiratory Society·Isabelle Sermet-Gaudelus
Mar 23, 2013·The European Respiratory Journal·Stephen StickFelix Ratjen
Apr 18, 2013·American Journal of Respiratory and Critical Care Medicine·Jane C DaviesUNKNOWN VX08-770-103 (ENVISION) Study Group
May 23, 2013·The New England Journal of Medicine·Peter D SlyUNKNOWN AREST CF Investigators
Jun 8, 2013·American Journal of Respiratory and Critical Care Medicine·Padmaja SubbaraoFelix Ratjen
Oct 1, 2013·American Journal of Respiratory Cell and Molecular Biology·Lydia C PowellDavid W Thomas
Oct 31, 2013·American Journal of Respiratory and Critical Care Medicine·Ryan J AdamDavid A Stoltz
May 20, 2014·The Lancet. Respiratory Medicine·Eitan KeremUNKNOWN Cystic Fibrosis Ataluren Study Group
Nov 5, 2014·Chest·Marcus P Kennedy, Barry J Plant

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Jun 4, 2019·International Journal of Pediatric Otorhinolaryngology·P SuyS Ayari-Khalfallah

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