Current Research Studies
A Post-Authorization Safety Study (PASS) to Assess Long-term Safety in Patients with Pompe Disease Treated with Avalglucosidase alfa in the Real-World Setting: the SAVANT (Safety assessment of AValglucosidase Alfa as a Novel Treatment) study
Safety assessment of AValglucosidase Alfa
What is the goal of the study?
To collect long term safety data and evaluate the important identified risk of infusion associated reactions (IARs) including hypersensitivity and anaphylactic reactions and the important potential risk of medication errors in participants treated with avalglucosidase alfa in both clinic/hospital and/or home settings as per standard of care (SoC) determined by their treating physician. In addition, to collect and evaluate the immunogenicity of avalglucosidase alfa treatment in the long-term.
Who can participate in the study?
Please contact the study team listed below to learn more.