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Gaucher DiseaseRare disease

What this study is

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.

Study details

Sponsor
Shire
Conditions
Gaucher Disease
Enrolling
1,257 people
Starts
2010-12-29
Est. completion
2028-09-30
Record
View on ClinicalTrials.gov ↗