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Updated: Oct 28, 2025

An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
Published on: October 21, 2014
Marc Engelen1, Stephan Kemp2, Florian Eichler3
1Department of Pediatric Neurology, Emma Children's Hospital, and Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.
X-linked adrenoleukodystrophy (ALD) is a genetic disorder affecting fatty acid metabolism. Treatments like hematopoietic cell transplantation can halt leukodystrophy, but myelopathy remains untreatable.
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