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GAUCHER, fabry, mps i, and pompe registries

Summary:

Rare Disease Registries for Lysosomal Storage Disorders

Other Studies:

CARAT – EFC 16158

Recruiting, Fabry Disease

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

Hypophosphatasia, Recruiting

Mulberry -Alexion 1850-305

Hickory -Alexion 1850-301

Hypophosphatasia, Recruiting

Hickory - Alexion 1850-301

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