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A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
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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.
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 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
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
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
Longitudinal Investigation of Neurodegenerative Disorders in Children
*Original research initiated by investigators at UPMC Children’s Hospital of Pittsburgh.
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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