Cystic fibrosis (CF) is a genetic disorder caused by a mutation
in the gene that makes the cystic fibrosis transmembrane
conductance regulator (CFTR) protein. A specific type of
mutation called a nonsense (premature stop codon) mutation is
the cause of CF in approximately 10% of subjects with the
disease. Ataluren (PTC 124) is an orally delivered
investigational drug that has the potential to overcome the
effects of the nonsense mutation. This study was a Phase 3 trial
that evaluated the clinical benefit of Ataluren (PTC124) in
adult and pediatric patients with CF due to a nonsense mutation.
The main goals of the study were to understand whether Ataluren
(PTC 124) can improve pulmonary function and whether the drug
can safely be given for a long period of time. The study also
assessed the effects of Ataluren (PTC 124) on CF pulmonary
exacerbation frequency, cough frequency, health-related quality
of life, antibiotic use for CF-related infections, CF-related
disruptions to daily living, body weight, and CF pat