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For more information about research, please call our main office at 412-692-6438.
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The Program for the Study of Neurodevelopment in Rare Disorders (NDRD) is committed to research that will further the understanding of rare neurodegenerative diseases with the goal of developing new treatments to help affected children live longer, healthier lives.
The research of the NDRD uses an interdisciplinary translational approach to answer key clinical questions. Among our protocols are:
A natural history study tracks the course of a disease over time in untreated patients. Rare diseases often have many forms, which differ in terms of age at onset, specific symptoms and their severity, and how rapidly the disease progresses. Identifying how the natural progression of rare neurodegenerative diseases affects the brain helps researchers evaluate the effectiveness of new treatments. Through an Institutional Review Board-approved longitudinal research study, the NDRD has developed a database containing detailed information from more than 600 evaluations of more than 325 patients with rare neurodegenerative disorders.
A core research initiative is the NDRD Neuroimaging Research Program, which aims to develop new imaging technologies that improve the understanding of early brain development and how it is affected by disease progression. The long-term outcomes for children with neurodegenerative diseases improve with early diagnosis and therapy. The NDRD neuroimaging team of clinicians, neuroradiologists, statisticians, and computer scientists is developing new stringent methodologies to analyze this complex data. The NDRD database has imaging data from more than 250 healthy children scanned as neonates and at 1, 2, 4, and 6 years of age. The database also includes over 300 scans of more than 160 children with genetic neurodegenerative diseases.
The Program for the Study of NDRD team works with pharmaceutical and biotech companies that are developing a diverse range of treatments for neurodegenerative diseases. Investigational treatments include enzyme replacement therapy, gene therapy, and umbilical cord blood transplantation. Correlating functional outcomes with neuroimaging findings with will guide the development and refinement of new treatments. The NDRD database contains over 1,000 detailed evaluations of more than 200 children who received umbilical cord stem cell transplantation.
Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 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 Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy
Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome
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
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121
Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects with MPS II (Hunter Syndrome)
Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Longitudinal Investigation of Neurodegenerative Disorders in Children
Michele Poe, PhD
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.
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