3 results
Approved WMOCompleted
1. A better understanding of the apraglutide dose/concentration-effect relationship;2. A better understanding of duration of effect (apraglutide kinetics in relation to citrulline kinetics).
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 WMORecruiting
The purpose of this study is to see if the study compound, apraglutide, has any effect on the gastric emptying of liquids in healthy subjects. Gastric emptying is the normal way in which food passes through the body, when the contents of the stomach…