Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251)

BACKGROUND: This is the first-in-human study of icenticaftor, an oral potentiator of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) channel. Restoration of CFTR activity has shown significant clinical benefits, but more studies are needed to address all CFTR mutations. METHODS:...

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Detalles Bibliográficos
Autores principales: Kazani, Shamsah, Rowlands, David J., Bottoli, Ivan, Milojevic, Julie, Alcantara, Jose, Jones, Ieuan, Kulmatycki, Kenneth, Machineni, Surendra, Mostovy, Lidia, Nicholls, Ian, Nick, Jerry A., Rowe, Steven M., Simmonds, Nicholas J., Vegesna, Raju, Verheijen, Jeroen, Danahay, Henry, Gosling, Martin, Ayalavajjala, Phaninatha Sarma, Salman, Mohammed, Strieter, Robert
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8935475/
https://www.ncbi.nlm.nih.gov/pubmed/33293212
http://dx.doi.org/10.1016/j.jcf.2020.11.002

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