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Found 247 clinical trials
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International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

To characterize the Gaucher disease population. To evaluate the long-term effectiveness of imiglucerase and of eliglustat. Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy.

gaucher disease
imiglucerase
eliglustat
deficiency
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  • 19 Feb, 2024
  • 209 locations
Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3

Primary Objectives: Part 1: Cohorts 1, 2 and 3 Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease (GD) Type 3 patients that distinguish GD3 from adult Gaucher disease Type 1 (GD1) patients. Screen adult and pediatric GD3 patients who qualify for treatment with venglustat in Parts 2 and 3.

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  • 19 Feb, 2024
  • 7 locations