3 results
Primary objective- Survival without BPD at 36 weeks gestational age (GA)Secondary objectives- Neurodevelopment at a corrected age of 18-22 months- Adverse treatment effects- Mortality at 36 weeks gestational age- BPD incidence at 36 weeks…
Primary objectiveTo evaluate the long-term safety of 10-, 10-, 20-mg/kg ataluren in patients with nonsense mutation cystic fibrosis (nmCF), who previously participated in pivotal study PTC124-GD-021-CF, as determined by adverse events and laboratory…
To evaluate the ability of ataluren to improve pulmonary function relative to placeboTo determine the effect of ataluren on:1. Pulmonary symptoms2. General well-being3. Health-related Quality Life (HRQL)