3 results
Not approvedWill not start
1.3. Study Design Rationale The ATLAS-INH trial (ALN-AT3SC-003) is a multicenter, multinational, randomized, openlabel Phase 3 study designed to demonstrate the efficacy and safety of fitusiran in patients with haemophilia A or B with inhibitory…
Approved WMOWill not start
To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.
Approved WMORecruiting
The primary objective of the UNLOAD-CHIP is to investigate the combined clinical endpoint (as described in primary and secundary endpoints).