Gaucher Disease Enzyme Replacement Therapy Registry

Active Surveillance Taliglucerase Alfa Registry in Patients with Gaucher Disease

Protocol Description

The purpose of this observational study is to assess the long-term effects of taliglucerase alfa (Elelyso®/Uplyso) enzyme replacement therapy as part of normal medical care by collecting and analyzing information about patients treated with this drug for Gaucher disease.

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting patients of both genders and of all ages who have a confirmed diagnosis of Gaucher disease.
Males and Females: All ages


Physicians must provide medical history information about their enrolled patients. Information from other tests required over time for disease management, including optional antibody testing, will be entered into the database.
Visits: Not applicable
Duration: 10 years

Status: Open to Enrollment

Source(s) of Support


Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC