Rare diseases Xagena
A study has identified a gene mutation that researchers estimate dates back to 11,600 B.C., making it the second oldest human disease mutation yet discovered.Researchers with the Ohio State University ...
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 ...
Office of Orphan Products Development ( OOPD ) of the Food and Drug Administration ( FDA ) has granted orphan drug designation for drug candidate, Erdosteine, for the treatment of bronchiectasis. Erdo ...
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 ...
Pseudomonas aeruginosa is the main pathogen in bronchopulmonary infections in cystic fibrosis ( CF ) patients. It can only be eradicated at early infection stages while reduction of its bacterial load ...
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 ...
