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Found 2 clinical trials
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (LEAP2MONO)  

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions

neuronopathic gaucher disease
deficiency
replacement therapy
bone pain
platelet count
  • 6 views
  • 13 May, 2022
  • 1 location
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (LEAP2MONO)

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions

neuronopathic gaucher disease
deficiency
replacement therapy
platelet count
bone pain
  • 0 views
  • 09 May, 2022
  • 1 location