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Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3
Cohort 3 Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing: CSF Lyso-GL1 levels Modified Friedreichs Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Brain resting-state functional Magnetic Resonance Imaging (rs-fMRI) reflecting connectivity between parieto-occipital areas Bone disease manifestations Evaluate safety …
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- 04 Dec, 2020
- 7 locations
