The Natural History of Infantile Globoid Cell Leukodystrophy

Inclusion Criteria: * Informed consent obtained before any study-related activities. (Study-related activities are any procedure that would not have been performed during normal management of the subject) * The patient must have a documented diagnosis of infantile globoid cell leukodystrophy with galactocerebroside ß-galactosidase (GALC) activity \< 0.50 nmol/h/mg protein and evidence of two pathogenic mutations in the GALC gene must be confirmed after the baseline visit * The patient must have an age at the time of screening \< 2 years * The patient's parent(s) and/or legal guardian must have the ability to comply with the clinical protocol Exclusion Criteria: * History of hematopoietic stem cell transplantation * Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or other medical condition * Presence of major congenital abnormality * Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the principal investigator, would preclude participation in the study * Use of any investigational product within 30 days prior to study enrollment or currently enrolled in another study which involves clinical investigations * The patient's parent(s) and/or legal guardian is unable to understand the nature, scope, and possible consequences of the study * Patient is unable to comply with the protocol, i.e. inability to return for follow-up evaluations or otherwise unlikely to complete the …