| Popis: |
Rationale: We previously reported that ivacaftor was safe and well tolerated in cohorts aged 12 to 3 to ≤5× the upper limit of normal at Week 24. No other adverse trends in laboratory tests, vital signs, or ECG parameters were reported. Sweat chloride concentrations and measures of pancreatic obstruction improved. Conclusions: This study of ivacaftor in the first year of life supports treating the underlying cause of cystic fibrosis in children aged ≥4 months with one or more gating mutations. Clinical trial registered with clinicaltrials.gov (NCT02725567). |
