2 results
Approved WMOCompleted
To investigate the safety, tolerability and behavior in the body (absorption and excretion) of oral intake of TMC589337 and TMC589354 after ascending single intakes. In addition, the safety, tolerability and behavior in the body after multipe dose…
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…