Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation
Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation
About this item
Full title
Author / Creator
Accurso, Frank J , Rowe, Steven M , Clancy, J.P , Boyle, Michael P , Dunitz, Jordan M , Durie, Peter R , Sagel, Scott D , Hornick, Douglas B , Konstan, Michael W , Donaldson, Scott H , Moss, Richard B , Pilewski, Joseph M , Rubenstein, Ronald C , Uluer, Ahmet Z , Aitken, Moira L , Freedman, Steven D , Rose, Lynn M , Mayer-Hamblett, Nicole , Dong, Qunming , Zha, Jiuhong , Stone, Anne J , Olson, Eric R , Ordoñez, Claudia L , Campbell, Preston W , Ashlock, Melissa A and Ramsey, Bonnie W
Publisher
Waltham, MA: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
Waltham, MA: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
In vitro studies have indicated that a molecule, VX-770, potentiates the activity of a mutant cystic fibrosis transmembrane conductance regulator (CFTR) protein. This trial involving persons with a copy of the mutant allele showed improvement in those who received the drug.
Cystic fibrosis is a progressive lung disease
1
caused by mutation...
Alternative Titles
Full title
Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation
Authors, Artists and Contributors
Author / Creator
Rowe, Steven M
Clancy, J.P
Boyle, Michael P
Dunitz, Jordan M
Durie, Peter R
Sagel, Scott D
Hornick, Douglas B
Konstan, Michael W
Donaldson, Scott H
Moss, Richard B
Pilewski, Joseph M
Rubenstein, Ronald C
Uluer, Ahmet Z
Aitken, Moira L
Freedman, Steven D
Rose, Lynn M
Mayer-Hamblett, Nicole
Dong, Qunming
Zha, Jiuhong
Stone, Anne J
Olson, Eric R
Ordoñez, Claudia L
Campbell, Preston W
Ashlock, Melissa A
Ramsey, Bonnie W
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3148255
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3148255
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa0909825
