Ontology highlight
ABSTRACT:
SUBMITTER: Engelen M
PROVIDER: S-EPMC3401223 | biostudies-literature | 2012
REPOSITORIES: biostudies-literature
Engelen Marc M Tran Luc L Ofman Rob R Brennecke Josephine J Moser Ann B AB Dijkstra Inge M E IM Wanders Ronald J A RJ Poll-The Bwee Tien BT Kemp Stephan S
PloS one 20120720 7
<h4>Unlabelled</h4>X-linked adrenoleukodystrophy (X-ALD) is caused by mutations in the ABCD1 gene and is characterized by impaired beta-oxidation of very-long-chain fatty acids (VLCFA) and subsequent VLCFA accumulation in tissues. In adulthood X-ALD most commonly manifests as a gradually progressive myelopathy, (adrenomyeloneuropathy; AMN) without any curative or disease modifying treatments. We recently showed that bezafibrate (BF), a drug used for the treatment of hyperlipidaemia, reduces VLCF ...[more]