Krabbe Disease Natural History Study

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A home-based, natural history study of infantile globoid cell leukodystrophy (Krabbe Disease). This study is being conducted to gather prospective data on disease progression in infants diagnosed with GLD. This study will be performed using protocol-defined, standardized assessments including clinical, developmental, and neurologic measures. All study visits will be conducted in the subject's home. No travel to the study site is necessary.

Contact information:

Agnes Chen, MD, PI

Assistant Clinical Professor of Pediatrics and Neurology

David Geffen School of Medicine at UCLA

Harbor-UCLA Medical Center

Division of Pediatric Neurology, Box 468

1000 West Carson Street

Torrance, CA 90509

Phone: 310-222-4160

Fax: 310-320-2271


The study was approved March 31, 2010, by the Human Subjects Committee at Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center.


Sponsored by Shire Human Genetic Therapies


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Last revised 02-Aug-2010 by Steven Leber