Krabbe Disease Natural History Study
Academy Sites Books Clinical CME E-mail Journals Links Medline Parents Pharmaceuticals Professional Research Teaching Web Portals Internet Course
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
Go back to Child-Neuro Homepage
Go back to Seeking Patients page
Last revised 02-Aug-2010 by Steven Leber