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.
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
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