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 WMOWill not start
Primary* To estimate the effect of NME combination therapies on inducing a functional cure over the control arm.Secondary * To characterize the efficacy profile of NME combination therapies.* To characterize the PD profile of NME combination…