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 purpose of this study is to assess the safety and potential effectiveness of using gene therapy for infants with Krabbe disease following hematopoietic stem cell transplantation (HSCT), the current standard of care. The drug FBX-101, also called AAVrh10, delivers a normal copy of the GALC gene to those cells in the child’s central and peripheral nervous systems that may not be addressed by transplant. The goal is to restore normal production of the galactocerebrosidase (GALC) enzyme, which is deficient in patients with Krabbe disease.
Subject to certain exclusion criteria, this study is enrolling infants from birth through 12 months of age who have been diagnosed with Krabbe disease. Males and Females: Birth through 12 months
Participants will be given chemotherapy about 10 days before HSCT, and a one-time dose of FBX-101 will be administered about 21 days later, including a 2-week hospital stay for monitoring. There will be 12 outpatient follow-up visits over the next 2 years to record progress of the disease and any side effects from treatment. Visits: About 13, plus hospitalization for HSCT and gene therapy Duration: About 26 months (2 years, 2 months)
Forge Biologics, Inc.
Study Description at National Institutes of Health Krabbe Disease Neurodevelopment in Rare Disorders Research
Randy Windreich, MD
For more information about the study or enrollment, please contact: Brie Yanniello, NDRD Program Coordinator 412-692-6350
*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.
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.