CARAT - EFC 16158Summary: This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate...
Mulberry -Alexion 1850-305
Hickory - Alexion 1850-301
The Canadian Fabry Disease Initiative Registry. Click here to learn more
GAUCHER, fabry, mps i, and pompe registriesSummary: Rare Disease Registries for Lysosomal Storage Disorders
A randomized, double-blind, placebo-controlled, 12-month Phase III study to evaluate the effect of Venglustat on neuropathic and abdominal pain in male and female adults with Fabry disease who are treatment-naive or untreated for at least 6 months