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In Vivo and In Vitro Ivacaftor Response in Cystic Fibrosis Patients With Residual CFTR Function: N-of-1 Studies

Rationale: Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, decreases sweat chloride concentration, and improves pulmonary function in 6% of cystic fibrosis (CF) patients with specific CFTR mutations. Ivacaftor increases chloride transport in many other CFTR mutat...

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Bibliografische gegevens
Gepubliceerd in:Pediatr Pulmonol
Hoofdauteurs: McGarry, Meghan E., Illek, Beate, Ly, Ngoc P., Zlock, Lorna, Olshansky, Sabrina, Moreno, Courtney, Finkbeiner, Walter E., Nielson, Dennis W.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2017
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5461115/
https://ncbi.nlm.nih.gov/pubmed/28068001
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ppul.23659
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