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
Objectives: The primary aim of this study is to provide evidence for the effect of ITB treatment on the level of activities in dystonic CP patients.