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.
The purpose of this multi-center global clinical research study is to see if the new compound DNL310, an enzyme replacement therapy (ERT) that is thought to penetrate the central nervous system (CNS), is safe and tolerable when given to patients with mucopolysaccharidosis type II (MPS II), also called Hunter syndrome. In this inherited genetic condition, a defective iduronate-2-sulfatase gene (IDS) prevents production of the enzyme iduronate-2-sulfatase (I2S). The lack of circulating I2S enzyme can lead to progressive damage to the brain, heart, and other organs. The study drug has been designed to deliver I2S to both the body and brain.
Subject to exclusion criteria, this study is enrolling male participants ages 2 through 18 years with a confirmed diagnosis of MPS II.
Males: Ages 2 through 18 years
During the treatment period, participants will receive weekly intravenous infusions of DNL310 at dose levels specified by the study protocol. Examinations and other assessments will also be done during study visits.
Visits: 17, including screening
Duration: 24 weeks
Denali Therapeutics Inc.
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.