2 results
Approved WMOCompleted
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…
Approved WMOCompleted
The purpose of this study is to identify the first time-point the OMERACT RAMRIS score for the activity of synovitis is statistically significantly reduced compared to Baseline, in response to cerolizumab pegol (CZP) therapy. In addition, the early…