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Sponsor: Genzyme, a Sanofi Company
Conditions: Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
Countries: United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Gaucher disease population. * To evaluate the long-term effectiveness of imiglucerase and of eliglustat. Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.
Sex: ALL
Healthy volunteers: No
Study type: OBSERVATIONAL
Inclusion Criteria: ICGG Gaucher Registry * All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented β-glucocerebrosidase deficiency and/or mutation in the β-glucocerebrosidase gene. * For all patients, appropriate patient authorization will be obtained. Gaucher Pregnancy Sub-registry: * be enrolled in the ICGG Gaucher Registry. * be pregnant, or have been pregnant with appropriate medical documentation available. * provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed. Exclusion Criteria: \- No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.
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- New Haven, Connecticut, United States
- Washington D.C., District of Columbia, United States
- Washington D.C., District of Columbia, United States
- Coral Springs, Florida, United States
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- Decatur, Georgia, United States
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- Iowa City, Iowa, United States
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