2 results
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…
Approved WMOCompleted
The main objective is to determine the efficacy of Lyumjev in the Inreda AP system. Secondary objectives are: to assess safety, differences in pharmacodynamics and differences in AP-related outcome measures.