M. Engelen PhD


M. Engelen PhD

Medical Specialist
Main activities
Patient care, Research
(pediatric) neurology
Focus of research

Peroxisomal metabolic disorders



The Department of Pediatric Neurology is a subdivision of the Department of Neurology and the Department of Pediatrics. The research program of the department is focused on clinical and translational research concerning leukodystrophies and neonatal neurology. These two main themes are imbedded in the nine translational research programs of Amsterdam Neuroscience, the research institute of neuroscience investigators in the Amsterdam area. All staff members of the Department of Pediatric Neurology are involved in research and collaborate within Neuroscience Amsterdam, with other academic and non-academic hospitals, and with international research groups.




As expertise center for leukodystrophies in The Netherlands, the department has a long tradition in clinical characterization of large cohorts of leukodystrophy patients, studies with advanced MRI techniques and gene finding studies. There is close collaboration with laboratory scientists. Cell and mouse models are used to study pathophysiology of leukodystrophies. Several clinical trials have been performed the past decade and it is our ambition to be a center for large multicenter international trials. There is specific emphasis on:


  1. Vanishing white matter and metachromatic leukodystrophy
  2. Hypomyelinating disorders like 4H syndrome and Pelizaeus Merzbacher disease
  3. Peroxisomal disorders, specifically X-linked adrenoleukodystrophy and Zellweger spectrum disorders
  4. Defects of white matter ion and water homeostasis



Key publications
  • Engelen Marc, Ofman Rob, Dijkgraaf Marcel G. W., Hijzen Michiel, van der Wardt Lucinda A., van Geel Bjorn M., de Visser Marianne, Wanders Ronald J. A., Poll-The Bwee Tien, Kemp Stephan Lovastatin in X-Linked Adrenoleukodystrophy New England journal of medicine 2010;362 (3):276-277 [PubMed]
  • Engelen Marc, Barbier Mathieu, Dijkstra Inge M. E., Schür Remmelt, de Bie Rob M. A., Verhamme Camiel, Dijkgraaf Marcel G. W., Aubourg Patrick A., Wanders Ronald J. A., van Geel Bjorn M., de Visser Marianne, Poll-The Bwee T., Kemp Stephan X-linked adrenoleukodystrophy in women: a cross-sectional cohort study Brain 2014;137 (Part 3):693-706 [PubMed]
  • Engelen Marc, Kemp Stephan, de Visser Marianne, van Geel Björn M., Wanders Ronald J. A., Aubourg Patrick, Poll-The Bwee Tien X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management Orphanet journal of rare diseases 2012;7 (1):51 [PubMed]
  • Engelen Marc, Schackmann Martin J. A., Ofman Rob, Sanders Robert-Jan, Dijkstra Inge M. E., Houten Sander M., Fourcade Stéphane, Pujol Aurora, Poll-The Bwee Tien, Wanders Ronald J. A., Kemp Stephan Bezafibrate lowers very long-chain fatty acids in X-linked adrenoleukodystrophy fibroblasts by inhibiting fatty acid elongation Journal of inherited metabolic disease 2012;35 (6):1137-1145 [PubMed]
  • Kemp Stephan, Huffnagel Irene C., Linthorst Gabor E., Wanders Ronald J., Engelen Marc Adrenoleukodystrophy - neuroendocrine pathogenesis and redefinition of natural history Nature reviews. Endocrinology 2016;12 (10):606-615 [PubMed]
All Publications
Research programmes

M. Engelen PhD (Translational studies in (peroxisomal) leukodystrophies)

S. Kemp PhD
Prof. B.T. Poll-The PhD

F.C.C. Klouwer

M.M.C. Voermans MSc

Medewerkers buiten research programmes van afdeling Neurologie

S. Kemp PhD (Lipid metabolism and Neurotoxicity)

Current research funding
  • AMC
  • Bluebird Bio, Inc.
  • Stichting AMC Foundation
  • SwanBio Therapeutics
  • Vertex Pharmaceuticals Inc.
  • ZonMw