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
Eli Kids EFC13738 Summary: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients
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ORBIT Study - UX143Summary: A clinical trial for children, adolescents, and young adults living with osteogenesis imperfecta (OI)
AT1001-030Summary: followME is a disease registry that will track the health of people living with Fabry disease over time. The goal is to evaluate how different types of disease management affect people living with Fabry.
SYNB1934-CP-003Summary: The goal of this new study is to evaluate an investigational drug called SYNB1934.
FACTSSummary: Phase I gene therapy for Fabry Disease
GTX-102Summary: A phase 1/2 open-label, multiple-dose, dose-escalating clinical trial of the safety and tolerability of GTX-102 in pediatric patients with Angelman Syndrome (AS)