Abstract
Since 2012, there is now causal treatment available for patients with cystic fibrosis who have a 3rd class mutation G551D. The mechanism of action of the drug Ivacaftor is based on repairing the function of the CFTR chloride channel, damaged by this mutation.
The confirmed efficacy of the new therapeutic modality, represented by Ivacaftor, brings a real hope that in due time it will be possible to offer causal treatment to patients with other types of mutations to the CFTR gene.