Niemann Pick disease type A/B

Non-industry sponsored (local) studies:

• Biomarkers in Niemann Pick disease
• Lipid profiles in Niemann Pick disease
• Secondary pathophysiology in Fabry disease, Gaucher disease and ASMD

Multicenter clinical trials the AMC participates in:

• Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (ASCEND)