COVID-19 Information 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 goal of this observational study is to collect medical information about people worldwide with the genetic disorder mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome, to better understand what happens to them over time. People with MPS VI lack an enzyme that normally breaks down a certain type of sugar, leading to its accumulation in body tissues causing physical problems such as decreased growth, nose, throat and ear problems, heart disease, bowel problems, and bone and joint problems. Through participation in this program, patient test results will be entered into a database along with other MPS VI patients. Analysis by the program sponsor may lead to insights that will help physicians learn more about treating people with the condition.
The study is accepting patients of both genders and of all ages who have a confirmed diagnosis of MPS VI. Males and Females: All ages
Physicians must provide information from a physical exam, a blood test, and a urine test for their enrolled patients. For participants who are also receiving enzyme replacement therapy, additional blood work may optionally be collected for analysis and entered into the study database. Information from other tests required by participants’ physicians over time for disease management will also be entered into the database. Visits: Not applicable Duration: Ongoing
BioMarin Pharmaceutical
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 412-692-6065
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.