COVID-19 Vaccine Information and Updates Read the Latest
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 objective of this international multi-center study is to see if the drug pegzilarginase can help people with arginase 1 deficiency. Arginase 1 deficiency is a rare disease caused by a defect in a specific gene that causes a deficiency of the enzyme called arginase 1 in the body. This enzyme’s job is to help break down the amino acid arginine. When arginase is not working, arginine can build up in the blood and can lead to serious effects on growth, health, and intellect.
Subject to certain exclusion criteria, candidates must have a confirmed diagnosis of arginase 1 deficiency. Males and Females: Ages 2 years and older
Participants will be randomly assigned to a group that receives either a placebo or the study drug via intravenous infusion, weekly for 24 weeks. After that period all participants will receive weekly infusions of pegzilarginase for an additional 150 weeks along with individualized disease management. Visits: About 178 Duration: About 3-1/2 years
Aeglea BioTherapeutics
Study Description at National Institutes of Health Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact: Jessica Lindenberger, MSN, CRNP, FNP-C 412-692-7530
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.