4 results
Primary Objective:* To compare event-free survival (EFS) between AG-120 + azacitidine and placebo + azacitidine. Key Secondary Objectives:* To compare the complete remission (CR) rate between AG-120 + azacitidine and placebo + azacitidine. EFS is…
The study will investigate the safety and efficacy of emicizumab in patients with hemophilia A with inhibitors against FVIII.
This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in patients of all ages with mild (FVIII level between > 5% and < 40%) or moderate hemophilia A (FVIII level between >= 1% and…
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…