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:* To assess the safety and tolerability of LAM-001 administered daily by dry powder inhaler (DPI) for 14 days in patients with lymphangioleiomyomatosis (LAM) (Period 1).* To assess the longer-term safety and tolerability of LAM-001…