Cystic fibrosis transmembrane conductance regulator biomarkers in 'real life': can we evaluate individual efficacy of cystic fibrosis transmembrane conductance regulator therapy?
نویسندگان
چکیده
Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, is approved for the treatment of patients with cystic fibrosis (CF) with the G551D mutation aged 6 years or older. To evaluate the efficacy of this CFTR-modulating therapy (CFTR-MT) biomarkers such as sweat chloride (SC), nasal potential difference (NPD) and intestinal current measurement (ICM) have been implemented.
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ورودعنوان ژورنال:
- Therapeutic advances in respiratory disease
دوره 9 4 شماره
صفحات -
تاریخ انتشار 2015