Rare diseases Xagena
Ivacaftor ( Kalydeco ) has shown a clinical benefit in patients with cystic fibrosis who have the G551D-CFTR mutation and reduced lung function. Lung clearance index ( LCI ) using multiple-breath wash ...
The European Commission has approved Kalydeco ( Ivacaftor ) for people with cystic fibrosis ages 6 and older who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane condu ...
Results from two phase 3 studies of the Tezacaftor ( VX-661 ) / Ivacaftor combination treatment that showed statistically significant improvements in lung function ( percent predicted forced expirator ...