Cargando…
Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy
Lumacaftor-ivacaftor is a combination of two small molecule therapies targeting the basic defect in cystic fibrosis (CF) at a cellular level. It is a precision medicine and its effects are specific to individuals with two copies of the p.Phe508del gene mutation. The drug combination works by restori...
| Autor principal: | |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Dove
2019
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6650604/ https://www.ncbi.nlm.nih.gov/pubmed/31409974 http://dx.doi.org/10.2147/DDDT.S153719 |