C26:0-Carnitine Is a New Biomarker for X-Linked Adrenoleukodystrophy in Mice and Man.

@article{Beek2016C260CarnitineIA,
  title={C26:0-Carnitine Is a New Biomarker for X-Linked Adrenoleukodystrophy in Mice and Man.},
  author={Malu-Clair van de Beek and Inge M. E. Dijkstra and Henk van Lenthe and Rob Ofman and Dalia Goldhaber-Pasillas and Nicolas Schauer and Martin J. A. Schackmann and Joo-Yeon Engelen-Lee and Fr{\'e}d{\'e}ric M Vaz and Wim Kulik and Ronald J. A. Wanders and Marc Engelen and Stephan Kemp},
  journal={PloS one},
  year={2016},
  volume={11 4},
  pages={e0154597}
}
X-linked adrenoleukodystrophy (ALD), a progressive neurodegenerative disease, is caused by mutations in ABCD1 and characterized by very-long-chain fatty acids (VLCFA) accumulation. Virtually all males develop progressive myelopathy (AMN). A subset of patients, however, develops a fatal cerebral demyelinating disease (cerebral ALD). Hematopoietic stem cell transplantation is curative for cerebral ALD provided the procedure is performed in an early stage of the disease. Unfortunately, this narrow… CONTINUE READING
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