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 WMOWill not start
The purpose of this study is to see if Crinecerfont is effective in reducing daily glucocorticoid dosage while maintaining adrenal androgen control.The purpose of the study is also to see if Crinecerfont is effective in reducing adrenal steroid…