A Retrospective Study to Characterize the Natural History of Childhood Cerebral X-Linked Adrenoleukodystrophy and to Investigate the Influence of Allogeneic Transplantation on Affected Subjects

Principal Investigator: Gerald Raymond

This is a retrospective study designed to:

  1. Collect specific and sufficient data necessary to characterize the natural history of disease in subjects with untreated childhood cerebral ALD
  2. To collect efficacy and safety date from childhood cerebral ALD subjects that were treated with hematopoietic cell transplantation (HCT)