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VX-770, a CFTR potentiator, may have a potential clinical benefit in a subgroup of people with cystic fibrosis
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Cystic fibrosis (CF) is caused by mutations in the gene encoding the CFTR (CF transmembrane conductance regulator) protein, an epithelial ion channel. Some mutations, of which the G551D mutation is the most common (occurring in approximately 5% of people with CF), permit the defective CFTR protein to reach the epithelial …