Profile of tezacaftor/ivacaftor combination and its potential in the treatment of cystic fibrosis.
Ther Clin Risk Manag
; 15: 1029-1040, 2019.
Article
en En
| MEDLINE
| ID: mdl-31692517
ABSTRACT
Cystic fibrosis (CF) is a life-limiting autosomal recessive disease caused by dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel. Management of CF has traditionally relied upon managing complications of CFTR protein dysfunction and this has led to a steady improvement in survival of CF patients. However, the landscape of CF care has changed substantially over the last decade with the discovery of CFTR modulators that aim to increase or potentially restore the function of the disease-causing CFTR protein. This narrative review summarizes the development of CFTR therapies so far with emphasis on tezacaftor/ivacaftor combination therapy. We have also summarized the Phase II results of triple combination therapy which promises an effective CFTR modulator therapy for more than 90% of CF patients.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Idioma:
En
Revista:
Ther Clin Risk Manag
Año:
2019
Tipo del documento:
Article
País de afiliación:
Reino Unido