2 results
Approved WMOCompleted
The study will be performed in 3 parts, Parts 1, 2 and 3. In all parts, the purpose is to investigate to what extent APX001 is tolerated. In addition, it will be investigated how quickly and to what extent APX001 is absorbed and eliminated from theā¦
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.