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The purpose of this international multi-center study is to collect and analyze data about how an intravenous infusion of taliglucerase alfa (Elelyso™) in children with Type 1 Gaucher disease is processed by the body. Elelyso is approved in United States, Canada, Mexico, and other countries, and is available by prescription for Type 1 Gaucher disease. The manufacturer is seeking additional information about it so as to provide the best information to other patients with Gaucher disease in the future.
Subject to certain exclusion criteria, the study is accepting patients of both genders up to age 12 who have a confirmed diagnosis of Type 1 Gaucher disease and are already participating in the Gaucher Disease Enzyme Replacement Therapy Registry.
Boys and Girls: Up to age 12
Participants will be seen by the team for a screening visit to determine eligibility, a baseline visit to collect additional information about the child’s Gaucher disease, and additional visits to receive study medications and for blood sampling and other tests.
Duration: 13 months
Study Description at National Institutes of Health
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
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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