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Ivacaftor: A Novel Gene-Based Therapeutic Approach for Cystic Fibrosis
Ivacaftor is a new therapeutic agent that acts at the cystic fibrosis transmembrane conductance regulator (CFTR) channel to alter activity. It is approved for use in patients 6 years and older with cystic fibrosis who have at least 1 G551D mutation in the CFTR gene. It is unlike any other current ph...
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| Hauptverfasser: | , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Pediatric Pharmacy Advocacy Group
2013
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3626070/ https://ncbi.nlm.nih.gov/pubmed/23616732 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.5863/1551-6776-18.1.8 |
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