3 results
Approved WMOCompleted
2.5 Study rationaleThe nephrotoxicity caused by cisplatin is of great concern both in that it is dose-limiting and can be detrimental to the patient. Much work has been done to reduce or prevent this damage to renal cells, however to date the best…
Approved WMOCompleted
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…
Approved WMOCompleted
The study will investigate the safety and efficacy of emicizumab in patients with hemophilia A with inhibitors against FVIII.