Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis

Background Cystic fibrosis is a common life‐shortening genetic disorder in the Caucasian population (less common in other ethnic groups) caused by the mutation of a single gene that codes for the production of the cystic fibrosis transmembrane conductance regulator protein. This protein coordinates...

Ausführliche Beschreibung