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The purpose of this observational study is to assess the long-term effects of taliglucerase alfa (Elelyso®/Uplyso) enzyme replacement therapy as part of normal medical care by collecting and analyzing information about patients treated with this drug for Gaucher disease.
Subject to certain exclusion criteria, the study is accepting patients of both genders and of all ages who have a confirmed diagnosis of Gaucher disease.
Males and Females: All ages
Physicians must provide medical history information about their enrolled patients. Information from other tests required over time for disease management, including optional antibody testing, will be entered into the database.
Visits: Not applicable
Duration: 10 years
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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Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.