Article
Treatment of Cystic Fibrosis Patients Homozygous for F508del with Lumacaftor-Ivacaftor (Orkambi®) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells.
International journal of molecular sciences - 31 Mar 2020
Favia Maria, Gallo Crescenzio, Guerra Lorenzo, De Venuto Domenica, Diana Anna, Polizzi Angela Maria, Montemurro Pasqualina, Mariggiò Maria Addolorata, Leonetti Giuseppina, Manca Antonio, Casavola Valeria, Conese Massimo
Abstract excerpt
The treatment of cystic fibrosis (CF) patients homozygous for the F508del mutation with Orkambi®, a combination of a corrector (lumacaftor) and a potentiator (ivacaftor) of the mutated CFTR protein, resulted in some amelioration of the respiratory function. However, a great variability in the clinical response was also observed. The aim of this study was to evaluate the response to Orkambi® in a small cohort of...
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