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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...
Tallennettuna:
| Julkaisussa: | Drug Des Devel Ther |
|---|---|
| Päätekijä: | |
| Aineistotyyppi: | Artigo |
| Kieli: | Inglês |
| Julkaistu: |
Dove
2019
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| Aiheet: | |
| Linkit: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6650604/ https://ncbi.nlm.nih.gov/pubmed/31409974 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S153719 |
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