2 results
Approved WMOCompleted
To evaluate the proportion of patients indicating an overall preference via a Patient Preference Questionnaire (PPQ) for either the subcutaneous (SC) or the intravenous (IV) route of rituximab administration.
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…