2 results
Approved WMOCompleted
Single dose:The objectives of Part 1 (single dose escalation part) are to examine the safety, tolerability and pharmacokinetics (i.e., the circulating levels of TMC558445 in your blood over time) of increasing single oral doses of TMC558445, with…
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…