Investigational Site Number :380001

Udine 33100

3 studies enrolling now · 7 studies all time

What they study most

Cerebroside Lipidosis Syndrome, Fabry Disease, Gaucher Disease, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Glycogen Storage Disease Type II, Pompe Disease, Sphingomyelin Lipidosis

Studies at this site

• Fabry Disease Registry & Pregnancy Sub-registry — Recruiting now
• Pompe Disease Registry Protocol — Recruiting now
• International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry — Recruiting now
• Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency — Completed
• A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency — Completed
• A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD) — Completed
• Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency — Completed