3 results
Approved WMOPending
The primary objective is to evaluate the effect of a once-a-weekprophylaxis regimen with BAY 79-4980 on the protection from all bleeds compared to a three times-per-week prophylaxis regimen with rFVIII-FSWFI.
Approved WMOCompleted
Primary:* To assess the efficacy of idebenone compared to placebo, in delaying the loss of respiratory function in patients with DMD receiving glucocorticoid steroids as measured by changes in Forced Vital Capacity percent predicted (FVC %p) using…
Approved WMOCompleted
To evaluate the efficacy, safety, tolerability, in subjects with ALS following the oral administration of study drug (active or placebo capsules).