Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy
Status:
No longer available
Trial end date:
1969-12-31
Target enrollment:
Participant gender:
Summary
X-linked adrenoleukodystrophy (ALD) is a genetic disorder affecting the brain and adrenal
glands. Approximately one third of boys who are at risk will develop cerebral disease. Using
a specific diet and the compound Lorenzo's oil, it can be shown that very long chain fatty
acids may be lowered in the blood, but it is not known to what degree that may prevent the
onset of childhood disease.
This proposal makes available Lorenzo's oil to individuals with ALD, a life threatening
disorder for which there are presently no other therapies.
Details
Lead Sponsor:
University of Minnesota University of Minnesota - Clinical and Translational Science Institute