Gaucher Disease Outcome Survey (GOS)

Brief description of study

Gaucher disease is one of several rare lysosomal storage disorders, affects 10,000-30,000 patients worldwide, and is classified into three clinical subtypes. Type 1 Gaucher disease is the most common type. It commonly affects the liver, spleen, bone marrow and rarely the kidneys. The brain is not affected. Type II disease begins within 6 months of birth and affects about half as many individuals as type 1 disease. In addition to involvement of the liver, spleen and bone marrow, the brain is affected causing brain damage and usually death by the age of 2 years. Type III Gaucher disease can occur at any time in childhood or adulthood. It is characterized by chronic and slowly progressive symptoms involving the liver, spleen and bone marrow and the nervous system. Patients with this type of disease usually live into adulthood. The objective of the study is to learn the natural history of the disease as well as the disease response to various treatments, to monitor the safety and effectiveness of treatment with velaglucerase alfa enzyme (VPRIV®) which is approved for Type I Gaucher disease, and other treatments, to learn more about the quality of life of patients with Gaucher disease and to provide information to the regulators who require ongoing information on the safety and effectiveness of VPRIV®.

Clinical Study Identifier: 11-01170 Identifier: NCT03291223
Other Investigator: Berrin Monteleone.

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