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
To evaluate LFS after allo HCT in AML/RAEB in complete remission using matched or unrelated donors in comparison to conventional chemotherapy