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This study is being done to help researchers better understand infantile globoid leukodystrophy (GLD), or Krabbe disease, a rare, inherited degenerative disorder that affects the nervous system and for which there is presently no cure. The study will gather information about participants over 18 months to document the progression of the disease. Relatively little is known about infantile GLD and no prior studies have examined its natural history, or progression, over an 18-month period. By better understanding parameters and progression of infantile GLD, researchers will lay a foundation for evaluation of potential future treatments developed for the disease.
Participants in this study include children of both genders, ages infant to 2 years, who have been diagnosed with GLD.
Boys and Girls: Ages infant to 24 months
The study involves a series of evaluations at the NDRD clinic at Children’s Hospital of Pittsburgh of UPMC, including brain imaging and other tests, all of which are considered to be standard care for patients with Krabbe disease.
Visits: 7, each of which will occur over 1 or 2 days
Duration: 18 months
Leaflet for Study Candidates (PDF)
ACE BioSciences A/S, subsidiary of Zymenex Holding A/S
Study Description at National Institutes of Health
Maria L. Escolar, MD, MS
For more information about the study or enrollment, please contact:
Program Coordinator Mary Brannaman, MPA, BS
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
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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