2 results
Approved WMOCompleted
A study to investigate the safety, pharmacokinetics and pharmacodynamics of a new recombinant human factor VIIa in healthy males, compared to Novoseven.
Approved WMOPending
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia…