2 results
Approved WMORecruiting
The primary objectives of the study are to:Determine the OBD(s) and RP2D's) of BMF-219 monotherapy administered daily based on evaluation of all available PK/ PD, target engagement, safety, and tolerability data. Note that the OBD and RP2D may…
Approved WMORecruiting
The primary objective of this study is to evaluate the efficacy of olipudase alfa in adult patients with acid sphingomyelinase deficiency.