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For more information about research, please call our main office at 412-692-6438.
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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.
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
Shire Human Genetic Therapies
Study Description at National Institutes of Health Gaucher Disease Medical Genetics Research
Damara Ortiz, MD
For more information about the study or enrollment, please contact: Nadene Henderson, MS, LCGC 412-692-3475
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh One Children’s Hospital Way 4401 Penn Ave. Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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