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.
1.To investigate COVID-19 vaccine responses of the standard schedule of 2 vaccinations with the Moderna mRNA-1273 vaccine (primary schedule) in patients with hematologic diseases, in particular those patients who are considered immunocompromised,…