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Gaucher Disease Outcome Survey (GOS)

Who can join

All ages · All sexes

Inclusion Criteria:

* Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease
* Signed and dated written informed consent from the patient or, for patients aged \<18 years (\<16 years in the United Kingdom \[UK\]), their parent and/or legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients.

Exclusion Criteria:

\- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)

About the study

The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician.

The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.

Sponsor: Shire · Participants: 1,257 · Started: Dec 29, 2010

Contact the study team

• Shire Contact · Phone: +1 866 842 5335

Official record on ClinicalTrials.gov — NCT03291223

Locations in the U.S.

Conditions

• Gaucher Disease

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.