Your health can’t wait. Learn how we’re making our facilities safer and schedule your care now. Learn more.
Children's Hospital is part of the UPMC family.
Our Sites
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Ranked #9 Nationally by U.S. News & World Report.
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. Visits: 5 Duration: 13 months
Pfizer
Study Description at National Institutes of Health Gaucher Disease Medical Genetics Research
Gerard Vockley, MD, PhD
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.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.