3 results
The primary objective of this study is to assess the efficacy and safety of Genz-112638 compared with Cerezyme after 52 weeks of treatment in patients with Gaucher disease type 1 who have been stabilized with Cerezyme. The secondary objective of…
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.
To perform a double-blind randomized placebo controlled multicenter study with darbepoetin in infants with MRI confirmed PAIS and to investigate whether darbepoetin can reduce brain injury in neonates who suffered from perinatal arterial ischemic…