Gaucher Disease Outcome Survey – The GOS Study

An Observational, International, Multi-center, Long-Term Registry of Patients with Gaucher Disease

Protocol Description

The Gaucher Disease Outcome Survey (GOS) is an ongoing observational, international, multi-center, long-term database of patients with Gaucher Disease. GOS collects information from patients about their Gaucher disease progression, their medical management, and the use of current or past medications or treatments, if any. The principal aim of GOS is to use the data collected to better understand the natural history of the disease, how it is treated, its response to treatment, and possibly how to improve care for patients.

Eligibility Criteria

Patients of any age or gender with a confirmed diagnosis of Gaucher disease may participate in the study.
Males and Females: All ages


No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician, who is also responsible for recording relevant data into the GOS database.
Visits: None beyond normal clinical care
Duration: Ongoing

Status: Open to Enrollment

Source(s) of Support

Shire Human Genetic Therapies

Primary Investigator

Damara Ortiz, MD

Contact Information

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