Ivacaftor, a CFTR potentiator, improved lung function in certain cystic fibrosis patients (RCT)
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Most people with cystic fibrosis have mutations in the CFTR gene that prevent sufficient quantities of the assembled channels from making it to the cell membrane surface. That's a hard problem to fix. However, a small minority (4-5%) have mutations on the G551D allele that impair the function of the CFTR ion channel once it makes it to the membrane. Ivacaftor (VX-770) is an oral drug that somehow helps CFTR channels at the cell membrane stay open.
Ivacaftor, a CFTR potentiator, improved lung function in certain cystic fibrosis patients (RCT)
Ivacaftor, a CFTR potentiator, improved lung…
Ivacaftor, a CFTR potentiator, improved lung function in certain cystic fibrosis patients (RCT)
Most people with cystic fibrosis have mutations in the CFTR gene that prevent sufficient quantities of the assembled channels from making it to the cell membrane surface. That's a hard problem to fix. However, a small minority (4-5%) have mutations on the G551D allele that impair the function of the CFTR ion channel once it makes it to the membrane. Ivacaftor (VX-770) is an oral drug that somehow helps CFTR channels at the cell membrane stay open.