3 results
Approved WMOPending
To establish the safety, tolerability, and early efficacy of multiple doses of hLF1-11 given once daily for 14 days.
Approved WMOPending
Assess the neurological efficacy of ambroxol in adults and children with GD3.
Approved WMOWill not start
Primary Objective:* confirm the dose of nomacopan and sample size for Part B and evaluate the rank order of secondary endpointsOther Objectives:* compare nomacopan with adjunct OCS to placebo with adjunct OCS in achievement of Complete Remission of…