Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Royal Children's Hospital · Children's Medical Research Institute · +27 more institutions
Abstract
Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis.
through week 24 (calculated as a percentage) was a key secondary end point.
in favor of tezacaftor-ivacaftor over placebo were 4.0 percentage points and 6.8%, respectively (P
Citation impact
- FWCI
- 131.54
- Percentile
- 100%
- References
- 44
Authors
19- CWClaire WainwrightCorresponding
Royal Children's Hospital, Children's Medical Research Institute, Queensland Children’s Medical Research Institute, Children's Health Queensland Hospital and Health Service
- JEJ.S. Elborn
Queens University
- BWBonnie W. Ramsey
Seattle Children's Hospital, University of Washington
- GMGautham Marigowda
Vertex Pharmaceuticals (United States)
- XHXiaohong Huang
Vertex Pharmaceuticals (United States)
Topics & keywords
- Ivacaftor
- Medicine
- Cystic fibrosis
- Cystic fibrosis transmembrane conductance regulator
- Placebo
- Randomization
- Internal medicine
- Clinical endpoint
- Good health and well-being