Profile of tezacaftor/ivacaftor combination and its potential in the treatment of cystic fibrosis
| Autor: | Dejene Shiferaw, Shoaib Faruqi |
|---|---|
| Rok vydání: | 2019 |
| Předmět: |
congenital
hereditary and neonatal diseases and abnormalities Chemical Health and Safety Combination therapy biology business.industry General Medicine Disease respiratory system medicine.disease Bioinformatics Cystic fibrosis digestive system diseases Cystic fibrosis transmembrane conductance regulator respiratory tract diseases Ivacaftor medicine Tezacaftor Triple combination biology.protein Pharmacology (medical) General Pharmacology Toxicology and Pharmaceutics business Safety Research medicine.drug Cftr modulator |
| Zdroj: | Therapeutics and Clinical Risk Management. 15:1029-1040 |
| ISSN: | 1178-203X |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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