2 results
Approved WMOPending
The primary objective of this study is to confirm the efficacy and safety of Genz 112638 after 39 weeks of treatment in patients with Gaucher disease type 1.The secondary objective of this study is to determine the long term efficacy, safety, and…
Approved WMORecruiting
The primary objective of this study is: Part 1: To evaluate the efficacy of AL001 compared with placebo as measured by CDR® plus NACC FTLD-SB.Part 2: To assess the long-term safety and tolerability of AL001 in participants who have completed 96…