• Male

Dr. M. Engelen

Position: Other
Main activities: Patient care
Specialisation: Neurology (subspecialty: pediatric neurology)
foto
Focus of research:

Peroxisomal metabolic disorders

Leukodystrophies

 

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.

 

Leukodystrophies

 

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

 

 

AMC themes: Metabolic disorders and gastrointestinal diseases, Neurological & Psychiatric Disorders
Departments: Neurology, Paediatrics: Neurology
Braakman HMH, Engelen M, Nicolai J, Willemsen MAAP, Stroke mimics add to the phenotypic spectrum of GLUT1 deficiency syndrome. J NEUROL NEUROSUR PS 2017;ahead of print [PubMed]
Engelen M, Optimizing Treatment for Cerebral Adrenoleukodystrophy in the Era of Gene Therapy. NEW ENGL J MED 2017;377 (17):1682-1684 [PubMed]
Huffnagel IC, van de Beek MC, Showers AL, Orsini JJ, Klouwer FCC, Dijkstra IME, Schielen PC, van Lenthe H, Wanders RJA, Vaz FM, Morrissey MA, Engelen M, Kemp S, Comparison of C26:0-carnitine and C26:0-lysophosphatidylcholine as diagnostic markers in dried blood spots from newborns and patients with adrenoleukodystrophy. MOL GENET METAB 2017;ahead of print [PubMed]
Reijnders MRF, Janowski R, Alvi M, Self JE, van Essen TJ, Vreeburg M, Rouhl RPW, Stevens SJC, Stegmann APA, Schieving J, Pfundt R, van Dijk K, Smeets E, Stumpel CTRM, Bok LA, Cobben JM, Engelen M, Mansour S, Whiteford M, Chandler KE, Douzgou S, Cooper NS, Tan EC, Foo R, Lai AHM, Rankin J, Green A, Lönnqvist T, Isohanni P, Williams S, Ruhoy I, Carvalho KS, Dowling JJ, Lev DL, Sterbova K, Lassuthova P, Neupauerová J, Waugh JL, Keros S, Clayton-Smith J, Smithson SF, Brunner HG, van Hoeckel C, Anderson M, Clowes VE, Siu VM, Selber P, Leventer RJ, Nellaker C, Niessing D, Hunt D, Baralle D, PURA syndrome: clinical delineation and genotype-phenotype study in 32 individuals with review of published literature. J MED GENET 2017;-:- [PubMed]
van de Beek MC, Ofman R, Dijkstra I, Wijburg F, Engelen M, Wanders R, Kemp S, Lipid-induced endoplasmic reticulum stress in X-linked adrenoleukodystrophy. BBA-MOL BASIS DIS 2017;1863 (9):2255-2265 [PubMed]
van Haren K, Engelen M, Decision Making in Adrenoleukodystrophy When Is a Good Outcome Really a Good Outcome?. JAMA NEUROL 2017;74 (6):641-642 [PubMed]
Zeynelabidin S, Klouwer FCC, Meijers JCM, Suijker MH, Engelen M, Poll-The BT, van Ommen CH, Coagulopathy in Zellweger spectrum disorders: a role for vitamin K. J INHERIT METAB DIS 2017;ahead of print [PubMed]
Berendse K. Translational studies in Zellweger spectrum disorders. S.l.: s.n.; 2016. 221p. ISBN 978-94-028-0292-4 [UvA Dissertations Online] Proefschrift Universiteit van Amsterdam. (Promotor(s): Poll The BT, Wanders RJA; copromotor(s): Engelen M, Waterham HR)
Berendse K, Engelen M, Ferdinandusse S, Majoie CBLM, Waterham HR, Vaz FM, Koelman JHTM, Barth PG, Wanders RJA, Poll-The BT, Zellweger spectrum disorders: clinical manifestations in patients surviving into adulthood. J INHERIT METAB DIS 2016;39 (1):93-106 [PubMed]
Berendse K, Klouwer FCC, Koot BGP, Kemper EM, Ferdinandusse S, Koelfat KVK, Lenicek M, Schaap FG, Waterham HR, Vaz FM, Engelen M, Jansen PLM, Wanders RJA, Poll-The BT, Cholic acid therapy in Zellweger spectrum disorders. J INHERIT METAB DIS 2016;39 (6):859-868 [PubMed]
de Sain-van der Velden MGM, Jans JJ, Figee M, Engelen M, Prinsen HCMT, Verhoeven-Duif NM, van Kuilenburg ABP, Visser G, Vinkers CH, Stofwisselingsziekten binnen de psychiatrie. TIJDSCHR PSYCHIATR 2016;58 (5):402-406 [PubMed]
Hollak CEM, Biegstraaten M, Baumgartner MR, Belmatoug N, Bembi B, Bosch A, Brouwers M, Dekker H, Dobbelaere D, Engelen M, Groenendijk MC, Lachmann R, Langendonk JG, Langeveld M, Linthorst G, Morava E, Poll-The BT, Rahman S, Rubio-Gozalbo ME, Spiekerkoetter U, Treacy E, Wanders R, Zschocke J, Hagendijk R, Position statement on the role of healthcare professionals, patient organizations and industry in European Reference Networks. ORPHANET J RARE DIS 2016;11 (1):7 [PubMed]
Kemp S, Huffnagel IC, Linthorst GE, Wanders RJ, Engelen M, Adrenoleukodystrophy - neuroendocrine pathogenesis and redefinition of natural history. NAT REV ENDOCRINOL 2016;12 (10):606-615 [PubMed]
Klouwer FCC, Huffnagel IC, Ferdinandusse S, Waterham HR, Wanders RJA, Engelen M, Poll-The BT, Clinical and Biochemical Pitfalls in the Diagnosis of Peroxisomal Disorders. NEUROPEDIATRICS 2016;47 (4):205-220 [PubMed]
Koens LH, Kuiper A, Coenen MA, Elting JWJ, de Vries JJ, Engelen M, Koelman JHTM, van Spronsen FJ, Spikman JM, de Koning TJ, Tijssen MAJ, Ataxia, dystonia and myoclonus in adult patients with Niemann-Pick type C. ORPHANET J RARE DIS 2016;11 (1):121 [PubMed]
Menke LA, Engelen M, Alders M, Odekerken VJJ, Baas F, Cobben JM, Recurrent GNAO1 Mutations Associated With Developmental Delay and a Movement Disorder. J CHILD NEUROL 2016;31 (14):1598-1601 [PubMed]
Schackmann MJA, Ofman R, van Geel BM, Dijkstra IME, van Engelen K, Wanders RJA, Engelen M, Kemp S, Pathogenicity of novel ABCD1 variants: The need for biochemical testing in the era of advanced genetics. MOL GENET METAB 2016;118 (2):123-127 [PubMed]
van de Beek MC, Dijkstra IME, van Lenthe H, Ofman R, Goldhaber-Pasillas D, Schauer N, Schackmann M, Engelen-Lee JY, Vaz FM, Kulik W, Wanders RJA, Engelen M, Kemp S, C26:0-Carnitine Is a New Biomarker for X-Linked Adrenoleukodystrophy in Mice and Man. PLOS ONE 2016;11 (4):e0154597 [PubMed]
van der Land V, Mutsaerts HJMM, Engelen M, Heijboer H, Roest M, Hollestelle MJ, Kuijpers TW, Nederkoorn PJ, Cnossen MH, Majoie CBLM, Nederveen AJ, Fijnvandraat K, Risk factor analysis of cerebral white matter hyperintensities in children with sickle cell disease. BRIT J HAEMATOL 2016;172 (2):274-284 [PubMed]
Engelen M, Barbier M, Dijkstra IME, Schür R, de Bie RM, Verhamme C, Dijkgraaf MGW, Aubourg PA, Wanders RJA, van Geel BM, de Visser M, Poll-The BT, Kemp S, Reply: Age-dependent penetrance among females with X-linked adrenoleukodystrophy. BRAIN 2015;138 (2):e326 [PubMed]
Klouwer FCC, Berendse K, Ferdinandusse S, Wanders RJA, Engelen M, Poll-The BT, Zellweger spectrum disorders: clinical overview and management approach. ORPHANET J RARE DIS 2015;10:151 [PubMed]
van der Land V, Hijmans CT , de Ruiter M, Mutsaerts HJMM, Cnossen MH, Engelen M, Majoie CBLM, Nederveen AJ, Grootenhuis MA, Fijnvandraat K, Volume of white matter hyperintensities is an independent predictor of intelligence quotient and processing speed in children with sickle cell disease. BRIT J HAEMATOL 2015;168 (4):553-556 [PubMed]
van Geel BM, Poll-The BT, Verrips A, Boelens JJ, Kemp S, Engelen M, Hematopoietic cell transplantation does not prevent myelopathy in X-linked adrenoleukodystrophy: a retrospective study. J INHERIT METAB DIS 2015;38 (2):359-361 [PubMed]
van Schaik SM, Reneman L, Engelen M, Roos YBWEM, Poll-The BT, Strokelike episodes and cutis marmorata telangiectatica congenita. J CHILD NEUROL 2015;30 (1):129-132 [PubMed]
Velema M, Boot E, Engelen M, Hollak C, Parkinsonism in phenylketonuria: a consequence of dopamine depletion?. JIMD REPORTS 2015;20:35-38 [PubMed]
Berendse K, Engelen M, Linthorst GE, van Trotsenburg ASP, Poll-The BT, High prevalence of primary adrenal insufficiency in Zellweger spectrum disorders. ORPHANET J RARE DIS 2014;9:133 [PubMed]
de Beer M, Engelen M, van Geel BM, Frequent occurrence of cerebral demyelination in adrenomyeloneuropathy. NEUROLOGY 2014;83 (24):2227-2231 [PubMed]
Engelen M, Barbier M, Dijkstra IME, Schür R, de Bie RMA, Verhamme C, Dijkgraaf MGW, Aubourg PA, Wanders RJA, van Geel BM, de Visser M, Poll-The BT, Kemp S, X-linked adrenoleukodystrophy in women: a cross-sectional cohort study. BRAIN 2014;137 (Part 3):693-706 [PubMed]
Engelen M, Kemp S, Poll-The BT, X-linked adrenoleukodystrophy: pathogenesis and treatment. CURR NEUROL NEUROSCI 2014;14 (10):486 [PubMed]
Cobben JM, Engelen M, Polstra A, Array CGH on unstimulated blood does not detect all cases of Pallister-Killian syndrome: buccal smear analysis should remain the diagnostic procedure of first choice. AM J MED GENET A 2013;161AA (6):1517-1519 [PubMed]
Demirdas S, van Kessel IN, Korndewal MJ, Hollak CEM, Meutgeert H, Klaren A, van Rijn M, van Spronsen FJ, Bosch AM [Contributors: Asselbergs FW, Blank C, Derks TGJ, Diekman EF, Dijsselhof ME, Engelen M, van Hasselt PM, ter Horst NM, van den Hurk DAM, Janssen MCH, Karstens FPJ, van der Louw E, Morava E, Nicolai J, van de Pol L, Poll-The BT, Rubio-Gozalbo E, Smit GPA, de Ruijter J, Timmer C, Touw CML, Visser G, de Valk HW, Wijburg FA, Williams M]] , Clinical pathways for inborn errors of metabolism: warranted and feasible. ORPHANET J RARE DIS 2013;8 (1):37 [PubMed]
Engelen M, Ofman R, Dijkgraaf M, van Geel B, de Visser M, Wanders R, Poll-The BT, Kemp S, Comment on the paper "Effect of statin treatment on adrenomyeloneuropathy with cerebral inflammation: a revisit". CLIN NEUROL NEUROSUR 2013;115 (11):2401-2402 [PubMed]
Engelen M. Translational studies in X-linked adrenoleukodystrophy. s.l.: s.n.; 2012. 135p. ISBN 9789461914743 [UvA Dissertations Online] Proefschrift Universiteit van Amsterdam. (Promotor(s): Poll-The BT, de Visser M; copromotor(s): Kemp S, van Geel BM)
Engelen M, Kemp S, de Visser M, van Geel BM, Wanders RJA, Aubourg P, Poll-The BT, X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management. ORPHANET J RARE DIS 2012;7 (1):51 [PubMed]
Engelen M, Schackmann MJA, Ofman R, Sanders RJ, Dijkstra IME, Houten SM, Fourcade S, Pujol A, Poll-The BT, Wanders RJA, Kemp S, Bezafibrate lowers very long-chain fatty acids in X-linked adrenoleukodystrophy fibroblasts by inhibiting fatty acid elongation. J INHERIT METAB DIS 2012;35 (6):1137-1145 [PubMed]
Engelen M, Tran L, Ofman R, Brennecke J, Moser AB, Dijkstra IME, Wanders RJA, Poll-The BT, Kemp S, Bezafibrate for X-Linked Adrenoleukodystrophy. PLOS ONE 2012;7 (7):e41013 [PubMed]
Gevers S, Nederveen AJ, Fijnvandraat K, van den Berg SM, van Ooij P, Heijtel DF, Heijboer H, Nederkoorn PJ, Engelen M, van Osch MJ, Majoie CB, Arterial spin labeling measurement of cerebral perfusion in children with sickle cell disease. J MAGN RESON IMAGING 2012;35 (4):779-787 [PubMed]
Poll-The BT, Engelen M, Peroxisomal Leukoencephalopathy. SEMIN NEUROL 2012;32 (1):42-50 [PubMed]
Engelen M, van der Kooi AJ, Kemp S, Wanders RJA, Sistermans EA, Waterham HR, Koelman JTM, van Geel BM, de Visser M, X-linked adrenomyeloneuropathy due to a novel missense mutation in the ABCD1 start codon presenting as demyelinating neuropathy. J PERIPHER NERV SYST 2011;16 (4):353-355 [PubMed]
Engelen M, Westhoff D, de Gans J, Nederkoorn PJ, A 64-year old man presenting with carotid artery occlusion and corticobasal syndrome: a case report. J MED CASE REPORTS 2011;5 (1):357 [PubMed]
Poll The BT, Engelen M. X-gebonden adrenoleucodystrofie (X-ALD) in: Carbasius-Weber E, van Veen M, Visser G, editors. Dieet bij Metabole Ziekten. Utrecht: WKZ/UMC Utrecht; 2011., p. 278-289
Engelen M, Ofman R, Dijkgraaf MGW, Hijzen M, van der Wardt LA, van Geel BM, de Visser M, Wanders RJA, Poll-The BT, Kemp S, Lovastatin in X-Linked Adrenoleukodystrophy. NEW ENGL J MED 2010;362 (3):276-277 [PubMed]
Engelen M, Nederkoorn PJ, Smits M, van de Beek D, Delayed life-threatening subdural hematoma after minor head injury in a patient with severe coagulopathy: a case report. CASES J 2009;2:7587 [PubMed]
Engelen M, Kemp S, van Geel BM, Van gen naar ziekte; X-gebonden adrenoleukodystrofie. NED TIJDSCHR GENEESKD 2008;152 (14):804-808 [PubMed]
Engelen M, Ofman R, Mooijer PAW, Poll-The BT, Wanders RJA, Kemp S, Cholesterol-deprivation increases mono-unsaturated very long-chain fatty acids in skin fibroblasts from patients with X-linked adrenoleukodystrophy. BBA-MOL CELL BIOL L 2008;1781 (3):105-111 [PubMed]
de Jong BA, Engelen M, van Schaik IN, Vermeulen M, Confusing Cochrane reviews on treatment in multiple sclerosis. LANCET NEUROL 2005;4 (6):330-331 [PubMed]
Engelen M, Tijssen MAJ, Paroxysmal non-kinesigenic dyskinesia in antiphospholipid syndrome. MOVEMENT DISORD 2005;20 (1):111-113 [PubMed]
Boekholdt SM, Trip MD, Peters RJG, Engelen M, Boer JMA, Feskens EJM, Zwinderman AH, Kastelein JJP, Reitsma PH, Thrombospondin-2 polymorphism is associated with a reduced risk of premature myocardial infarction. ARTERIOSCL THROM VAS 2002;22 (12):E24-E27 [PubMed]
Docherty CC, Kalmar-Nagy J, Engelen M, Koenen SV, Nijland M, Kuc RE, Davenport AP, Nathanielsz PW, Effect of in vivo fetal infusion of dexamethasone at 0.75 GA on fetal ovine resistance artery responses to ET-1. AM J PHYSIOL-REG I 2001;281 (1):R261-R268 [PubMed]
Docherty CC, Kalmar-Nagy J, Engelen M, Nathanielsz PW, Development of fetal vascular responses to endothelin-1 and acetylcholine in the sheep. AM J PHYSIOL-REG I 2001;280 (2):R554-R562 [PubMed]
Key publications

Engelen M, Ofman R, Dijkgraaf MGW, Hijzen M, van der Wardt LA, van Geel BM, de Visser M, Wanders RJA, Poll-The BT, Kemp S, Lovastatin in X-Linked Adrenoleukodystrophy. NEW ENGL J MED 2010;362 (3):276-277 [PubMed]
Engelen M, Barbier M, Dijkstra IME, Schür R, de Bie RMA, Verhamme C, Dijkgraaf MGW, Aubourg PA, Wanders RJA, van Geel BM, de Visser M, Poll-The BT, Kemp S, X-linked adrenoleukodystrophy in women: a cross-sectional cohort study. BRAIN 2014;137 (Part 3):693-706 [PubMed]
Engelen M, Kemp S, de Visser M, van Geel BM, Wanders RJA, Aubourg P, Poll-The BT, X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management. ORPHANET J RARE DIS 2012;7 (1):51 [PubMed]
Engelen M, Schackmann MJA, Ofman R, Sanders RJ, Dijkstra IME, Houten SM, Fourcade S, Pujol A, Poll-The BT, Wanders RJA, Kemp S, Bezafibrate lowers very long-chain fatty acids in X-linked adrenoleukodystrophy fibroblasts by inhibiting fatty acid elongation. J INHERIT METAB DIS 2012;35 (6):1137-1145 [PubMed]
Kemp S, Huffnagel IC, Linthorst GE, Wanders RJ, Engelen M, Adrenoleukodystrophy - neuroendocrine pathogenesis and redefinition of natural history. NAT REV ENDOCRINOL 2016;12 (10):606-615 [PubMed]


All Publications

Research programmes

Dr. S. Kemp PhD (Lipid metabolism and Neurotoxicity)

Other research related activities
  • None reported
Current research funding
  • AMC
  • NIEUWE DEB IND
  • Stichting Stofwisselkracht
  • Stichting tot Steun EKZ-AMC
  • Vertex Pharmaceuticals Inc.
  • ZonMw