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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 Neurodevelopment in Rare Disorders (NDRD) team is currently conducting research on several diseases – disorders so rare, few physicians ever encounter them in their own practices.
Every patient is important in rare disease research. New and improved treatments are being advanced that may change outcomes for children and families living with these disorders. Participation in a research study can make an important difference in improving care for future generations.
All patients who participate in any of the research studies receive full clinical care and the benefit of the NDRD team’s experience, gained from evaluating hundreds of children with neurodegenerative diseases.
Some of the research opportunities related to NDRD are listed below. Select a study to review a brief description. Parents or guardians of children who might be eligible are encouraged to inquire about enrollment. Contact information is listed at the end of each study.
Randomized Study of Single vs. Multiple Privigen Dose Regimens in Pediatric CIDP
Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease
DTI as a Tool to Identify Infants with Krabbe Disease in Need of Urgent Treatment
The Natural History of Infantile Globoid Cell Leukodystrophy
Study of Intravenous Gene Transfer with an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation
Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
Natural History Study of Children with Metachromatic Leukodystrophy
Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome
Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction with Intravenous Elaprase® in Pediatric Patients with Hunter Syndrome and Cognitive Impairment
A Retrospective and Cross-sectional Study to Evaluate Neurodevelopmental Status in Pediatric Subjects with Severe Mucopolysaccharidosis Type II (Hunter Syndrome)
Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Subjects with Hunter Syndrome
Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects with MPS II (Hunter Syndrome)
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121
Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients with Middle and Advanced Phases of MPS IIIA Disease
Longitudinal Investigation of Neurodegenerative Disorders in Children
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
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