2 results
Approved WMOWill not start
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
Approved WMOCompleted
PRIMARY OBJECTIVE The primary objective of this study is to evaluate the effect of ALN-PCSSC treatment on LDL-C levels at Day 180. SECONDARY OBJECTIVESThe secondary objectives of this study are to evaluate the effect of ALN-PCSSC on the following…