Children's Hospital is part of the UPMC family.
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 #6 Nationally by U.S. News & World Report.
This is a long-term follow-up study to MPS II Treatment with RGX-121. No new treatment is provided through this study, as its purpose is to provide up to 3 years of observation and monitoring of effects and side-effects following gene-transfer provided in the initial study.
This study is open to those who participated in the base study. It commences at the termination of their involvement in the earlier research.
Boys: Ages 28 months to 7 years
Participants will have an initial enrollment visit, followed by annual visits with the study doctor for medical examinations, tests, and assessments. Additionally, parents/guardians may be contacted by the study team at the 6-month interval between visits for updates on the child’s health. The 3 years of visits and contacts adds to the initial 2 years of follow-up provided by the initial study, for a total patient monitoring period of up to 5 years.
Visits: 7, including contact calls
Duration: 3 years
Study Description at National Institutes of Health
Hunter Syndrome MPS II
Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
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.