M. Langeveld MD PhD publications

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Publications MD PhD M. Langeveld

Position
Medical Specialist
Main activities
Patient care, Research
Specialisation
Inherited metabolic disorders
Focus of research

My research focusses on inherited metabolic diseases in adults, with an emphasis on lysosomal storage disorders (LSDs) and fatty acid oxidation disorders (FAOD). LSD research is embedded in SPHINX, a collaboration of the departments of Radiology, Internal Medicine and Paediatrics and the Laboratory of Genetic and Metabolic Diseases of the AMC. Within the LDSs, my main focus is Fabry disease, I lead the following research projects, supervising 3 PhD students: 1. characterisation and consequences of the antibody response to enzyme replacement therapy (PhD student: Sanne van der Veen) 2. cognitive symptoms in Fabry disease and their relation to white matter abnormalities (PhD student: Simon Korver) 3. the natural history and consequences of cardiac pathology in Fabry disease (PhD student: Mohamed el Sayed). In addition I'm the local PI for international trials studying the safety and efficacy of a new form of enzyme replacement therapy. Recently, we launched an open access Fabry genotype phenotype website: www.fabrygenphen.com, providing open access to a large international database, simplifying interpretation of detected GLA gene variants. In my previous position at the Erasmus MC, I was the main supervisor of a PhD project on skeletal complications in two LSDs, the mucopolysaccharidosis and mucolipidosis (PhD student Esmee Oussoren).

In the field of fatty acid oxidation disorders, my research currently focusses on primary carnitine deficiency (collaboration with UMC Utrecht). PhD student Loek Crefcoeur performs studies on the natural history and the usefulness and feasibility of newborn screening for this disorder. 

Other research activities are a an exploratory study into the role the microbiome in urea cycle disorders and a project on discovery of inborn errors of metabolism in patients with an unexplained disorders (Zoektocht Erfelijke Metabole Aandoeningen (ZOEMBA) study.

I'm secretary of the adult metabolic physicians working group of the Society for Study of Inborn Errors of Metabolism (SSIEM, www.ssiem.org/amp) and as such involved in organising sessions at the yearly SSIEM conference and setting up a new Adult Inborn Errors of Metabolism training course. I’m an active member of the Internisten voor Erfelijke Stofwisselingsziekten (INVEST) group and coordinated the construction of the INVEST website (www.investof.nl) containing information on care of adults with inherited metabolic disorders in the Netherlands and Belgium (Vlaanderen). 

Amsterdam UMC Research Institutes
AMC departments
All publications

2021

  • Oussoren Esmee, Wagenmakers Margreet A. E. M., Link Bianca, van der Meijden Jan C., Pijnappel W. W. M. Pim, Ruijter George J. G., Langeveld Mirjam, van der Ploeg Ans T. Hip disease in Mucopolysaccharidoses and Mucolipidoses: A review of mechanisms, interventions and future perspectives Bone 2021;143 [PubMed]

2020

  • Körver Simon, Geurtsen Gert J., Hollak Carla E. M., van Schaik Ivo N., Longo Maria G. F., Lima Marjana R., Dijkgraaf Marcel G. W., Langeveld Mirjam Cognitive functioning and depressive symptoms in Fabry disease: a follow-up study Journal of inherited metabolic disease 2020;43 (5):1070-1081 [PubMed]
  • Körver Simon, Geurtsen Gert J., Hollak Carla E. M., van Schaik Ivo N., Longo Maria G. F., Lima Marjana R., Vedolin Leonardo, Dijkgraaf Marcel G. W., Langeveld Mirjam Depressive symptoms in Fabry disease: The importance of coping, subjective health perception and pain Orphanet journal of rare diseases 2020;15 (1) [PubMed]
  • Körver Simon, Longo Maria G. F., Lima Marjana R., Hollak Carla E. M., el Sayed Mohamed, van Schaik Ivo N., Vedolin Leonardo, Dijkgraaf Marcel G. W., Langeveld Mirjam Determinants of cerebral radiological progression in Fabry disease Journal of neurology, neurosurgery, and psychiatry 2020;91 (7):756-763 [PubMed]
  • van der Veen Sanne J., Hollak Carla E. M., van Kuilenburg André B. P., Langeveld Mirjam Developments in the treatment of Fabry disease Journal of inherited metabolic disease 2020;43 (5):908-921 [PubMed]
  • Knottnerus Suzan J. G., van Harskamp Dewi, Schierbeek Henk, Bleeker Jeannette C., Crefcoeur Loek L., Ferdinandusse Sacha, van Goudoever Johannes B., Houtkooper Riekelt H., IJlst Lodewijk, Langeveld Mirjam, Wanders Ronald J. A., Vaz Frédéric M., Wijburg Frits A., Visser Gepke Exploring the metabolic fate of medium-chain triglycerides in healthy individuals using a stable isotope tracer Clinical nutrition (Edinburgh, Scotland) 2020 [PubMed]
  • Korver Simon Fabry and the brain: Incorporating patients’ illness perceptions into the physicians’ practice 2020. 305p. ISBN 9789463757164. [UvA Dissertations Online] University of Amsterdam. (Supervisors: Hollak Carolina E. M., van Schaik Ivo N.; Co-supervisors: Langeveld Mirjam, Geurtsen Gert J.)
  • Benner Bernadette J. M., Bazelmans Mijke, Huidekoper Hidde, Langeveld Mirjam, Langendonk Janneke, Schoenmakers Sam Multidisciplinary approach in medicine: successful pregnancy in a patient with hyperinsulinism/hyperammonaemia (HI/HA) syndrome BMJ case reports 2020;13 (8) [PubMed]
  • Crefcoeur Loek L., de Sain-van der Velden Monique G. M., Ferdinandusse Sacha, Langeveld Mirjam, Maase Rose, Vaz Frédéric M., Visser Gepke, Wanders Ronald J. A., Wijburg Frits A., Verschoof-Puite Rendelien K., Schielen Peter C. J. I. Neonatal carnitine concentrations in relation to gestational age and weight JIMD reports 2020;56 (1):95-104 [PubMed]
  • Bleeker Jeannette C., Visser Gepke, Clarke Kieran, Ferdinandusse Sacha, de Haan Ferdinand H., Houtkooper Riekelt H., IJlst Lodewijk, Kok Irene L., Langeveld Mirjam, van der Pol W. Ludo, de Sain-van der Velden Monique G. M., Sibeijn-Kuiper Anita, Takken Tim, Wanders Ronald J. A., van Weeghel Michel, Wijburg Frits A., van der Woude Luc H., Wüst Rob C. I., Cox Pete J., Jeneson Jeroen A. L. Nutritional ketosis improves exercise metabolism in patients with very long-chain acyl-CoA dehydrogenase deficiency Journal of inherited metabolic disease 2020;43 (4):787-799 [PubMed]
  • van der Veen Sanne J., Vlietstra Wytze J., van Dussen Laura, van Kuilenburg André B. P., Dijkgraaf Marcel G. W., Lenders Malte, Brand Eva, Wanner Christoph, Hughes Derralynn, Elliott Perry M., Hollak Carla E. M., Langeveld Mirjam Predicting the development of anti-drug antibodies against recombinant alpha-galactosidase a in male patients with classical fabry disease International journal of molecular sciences 2020;21 (16):1-14 [PubMed]
  • Hollak Carla E. M., Sirrs Sandra, van den Berg Sibren, van der Wel Vincent, Langeveld Mirjam, Dekker Hanka, Lachmann Robin, de Visser Saco J. Registries for orphan drugs: generating evidence or marketing tools? Orphanet journal of rare diseases 2020;15 (1):235 [PubMed]
  • Knottnerus Suzan J. G., Bleeker Jeannette C., Ferdinandusse Sacha, Houtkooper Riekelt H., Langeveld Mirjam, Nederveen Aart J., Strijkers Gustav J., Visser Gepke, Wanders Ronald J. A., Wijburg Frits A., Boekholdt S. Matthijs, Bakermans Adrianus J. Subclinical effects of long-chain fatty acid β-oxidation deficiency on the adult heart: A case-control magnetic resonance study Journal of inherited metabolic disease 2020;43 (5):969-980 [PubMed]

2019

  • van der Veen S. J., van Kuilenburg A. B. P., Hollak C. E. M., Kaijen P. H. P., Voorberg J., Langeveld M. Antibodies against recombinant alpha-galactosidase A in Fabry disease: Subclass analysis and impact on response to treatment Molecular genetics and metabolism 2019;126 (2):162-168 [PubMed]
  • Sechi Annalisa, Fabbro Elisa, Langeveld Mirjam, Tullio Annarita, Lachmann Robin, Mochel Fanny Education and training in adult metabolic medicine: Results of an international survey JIMD reports 2019;49 (1):63-69 [PubMed]
  • Bleeker Jeannette C., Kok Irene L., Ferdinandusse Sacha, van der Pol W. Ludo, Cuppen Inge, Bosch Annet M., Langeveld Mirjam, Derks Terry G. J., Williams Monique, de Vries Maaike, Mulder Margot F., Gozalbo Estela R., de Sain-van der Velden Monique G. M., Rennings Alexander J., Schielen Peter J. C. I., Dekkers Eugenie, Houtkooper Riekelt H., Waterham Hans R., Pras-Raves Mia L., Wanders Ronald J. A., van Hasselt Peter M., Schoenmakers Marja, Wijburg Frits A., Visser Gepke Impact of newborn screening for very-long-chain acyl-CoA dehydrogenase deficiency on genetic, enzymatic, and clinical outcomes Journal of inherited metabolic disease 2019;42 (3):414-423 [PubMed]
  • Körver Simon, Feldt-Rasmussen Ulla, Svarstad Einar, Kantola Ilkka, Langeveld Mirjam Oral Chaperone Therapy Migalastat for the Treatment of Fabry Disease: Potentials and Pitfalls of Real-World Data Clinical pharmacology and therapeutics 2019;106 (5):925-926 [PubMed]
  • Körver Simon, Geurtsen Gert J., Hollak Carla E. M., van Schaik Ivo N., Longo Maria G. F., Lima Marjana R., Vedolin Leonardo, Dijkgraaf Marcel G. W., Langeveld Mirjam Predictors of objective cognitive impairment and subjective cognitive complaints in patients with Fabry disease Scientific reports 2019;9 (1) [PubMed]
  • Haijes Hanneke A., Molema Femke, Langeveld Mirjam, Janssen Mirian C., Bosch Annet M., van Spronsen Francjan, Mulder Margot F., Verhoeven-Duif Nanda M., Jans Judith J. M., van der Ploeg Ans T., Wagenmakers Margreet A., Rubio-Gozalbo M. Estela, Brouwers Martijn C. G. J., de Vries Maaike C., Langendonk Janneke G., Williams Monique, van Hasselt Peter M. Retrospective evaluation of the Dutch pre-newborn screening cohort for propionic acidemia and isolated methylmalonic acidemia: What to aim, expect, and evaluate from newborn screening? Journal of inherited metabolic disease 2019 [PubMed]
  • Hannah-Shmouni Fady, Stratakis Constantine A, Sechi Annalisa, Langeveld Mirjam, Hiwot Tarekegn G, Tchan Michel C, Mochel Fanny, Lynd Larry D, Sirrs Sandra Subspecialty training in adult inherited metabolic diseases: a call to action for unmet needs Lancet. Diabetes and endocrinology 2019;7 (2):82-84 [PubMed]
  • Körver Simon, van de Schraaf Sara A J, Geurtsen Gert J, Hollak Carla E M, van Schaik Ivo N, Langeveld Mirjam The Mini Mental State Examination does not accurately screen for objective cognitive impairment in Fabry Disease JIMD reports 2019;48 (1):53-59 [PubMed]

2018

  • Schuller Yvonne, Arends Maarten, Körver Simon, Langeveld Mirjam, Hollak Carla E. M. Adaptive pathway development for Fabry disease: a clinical approach Drug discovery today 2018;23 (6):1251-1257 [PubMed]
  • Langeveld M., Hollak C. E. M. Bone health in patients with inborn errors of metabolism Reviews in endocrine & metabolic disorders 2018;19 (1):81-92 [PubMed]
  • Langeveld Mirjam, Elstein Deborah, Szer Jeff, Hollak Carla E. M., Zimran Ari Classifying the additional morbidities of Gaucher disease Blood cells, molecules & diseases 2018;68:209-210 [PubMed]
  • Oussoren Esmee, Mathijssen Irene M. J., Wagenmakers Margreet, Verdijk Rob M., Bredero-Boelhouwer Hansje H., van Veelen-Vincent Marie-Lise C., van der Meijden Jan C., van den Hout Johanna M. P., Ruijter George J. G., van der Ploeg Ans T., Langeveld Mirjam Craniosynostosis affects the majority of mucopolysaccharidosis patients and can contribute to increased intracranial pressure Journal of inherited metabolic disease 2018;41 (6):1247-1258 [PubMed]
  • Körver Simon, Vergouwe Magda, Hollak Carla E. M., van Schaik Ivo N., Langeveld Mirjam Development and clinical consequences of white matter lesions in Fabry disease: a systematic review Molecular genetics and metabolism 2018;125 (3):205-216 [PubMed]
  • Langeveld M. Editorial commentary: Newborn screening for Fabry disease: Too much too soon? Trends in cardiovascular medicine 2018;28 (4):282-283 [PubMed]
  • Oussoren Esmee, van Eerd David, Murphy Elaine, Lachmann Robin, van der Meijden Jan C., Hoefsloot Lies H., Verdijk Rob, Ruijter George J. G., Maas Mario, Hollak Carla E. M., Langendonk Janneke G., van der Ploeg Ans T., Langeveld Mirjam Mucolipidosis type III, a series of adult patients Journal of inherited metabolic disease 2018;41 (5):839-848 [PubMed]
  • van Vliet Peter, Berden Annelies E., van Schie Mojca K. M., Bakker Jaap A., Heringhaus Christian, de Coo Irenaeus F. M., Langeveld Mirjam, Schroijen Marielle A., Arbous M. Sesmu Peripheral Neuropathy, Episodic Rhabdomyolysis, and Hypoparathyroidism in a Patient with Mitochondrial Trifunctional Protein Deficiency JIMD reports 2018;38:101-105 [PubMed]
  • van Karnebeek Clara D. M., Wortmann Saskia B., Tarailo-Graovac Maja, Langeveld Mirjam, Ferreira Carlos R., van de Kamp Jiddeke M., Hollak Carla E., Wasserman Wyeth W., Waterham Hans R., Wevers Ron A., Haack Tobias B., Wanders Ronald J. A., Boycott Kym M. The role of the clinician in the multi-omics era: are you ready? Journal of inherited metabolic disease 2018;41 (3):571-582 [PubMed]

2017

  • Oussoren Esmee, Bessems Johannes H. J. M., Pollet Virginie, van der Meijden Jan C., van der Giessen Lianne J., Plug Iris, Devos Annick S., Ruijter George J. G., van der Ploeg Ans T., Langeveld Mirjam A long term follow-up study of the development of hip disease in Mucopolysaccharidosis type VI Molecular genetics and metabolism 2017;121 (3):241-251 [PubMed]
  • Arends Maarten, Wijburg Frits A., Wanner Christoph, Vaz Frédéric M., van Kuilenburg André B. P., Hughes Derralynn A., Biegstraaten Marieke, Mehta Atul, Hollak Carla E. M., Langeveld Mirjam Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease Molecular genetics and metabolism 2017;121 (2):157-161 [PubMed]
  • Burgard Peter, Ullrich Kurt, Ballhausen Diana, Hennermann Julia B., Hollak Carla E. M., Langeveld Mirjam, Karall Daniela, Konstantopoulou Vassiliki, Maier Esther M., Lang Frauke, Lachmann Robin, Murphy Elaine, Garbade Sven, Hoffmann Georg F., Kölker Stefan, Lindner Martin, Zschocke Johannes Issues with European guidelines for phenylketonuria Lancet. Diabetes and endocrinology 2017;5 (9):681-683 [PubMed]
  • van Eerd D C D, Brussé I A, Adriaens V F R, Mankowski R T, Praet S F E, Michels M, Langeveld M Management of an LCHADD Patient During Pregnancy and High Intensity Exercise JIMD reports 2017;32:95-100 [PubMed]
  • Vroegindeweij Lena H P, Langendonk Janneke G, Langeveld Mirjam, Hoogendoorn Mels, Kievit Anneke J A, Di Raimondo Domenico, Wilson J H Paul, Boon Agnita J W New insights in the neurological phenotype of aceruloplasminemia in Caucasian patients Parkinsonism & related disorders 2017;36:33-40 [PubMed]
  • Langeveld Mirjam, Tan Chong Yew, Soeters Maarten R., Virtue Samuel, Watson Laura Pe, Murgatroyd Peter R., Ambler Graeme K., Vidal-Puig Santiago, Chatterjee Krishna V., Vidal-Puig Antonio No metabolic effects of mustard allyl-isothiocyanate compared with placebo in men American journal of clinical nutrition 2017;106 (5):1197-1205 [PubMed]
  • Biewenga M., Matawlie R. H. S., Friesema E. C. H., Koole-Lesuis H., Langeveld M., Wilson J. H. P., Langendonk J. G. Osteoporosis in patients with erythropoietic protoporphyria British journal of dermatology 2017;177 (6):1693-1698 [PubMed]

2016

  • Langeveld M., Tan C. Y., Soeters M. R., Virtue S., Ambler G. K., Watson L. P. E., Murgatroyd P. R., Chatterjee V. K., Vidal-Puig A. Mild cold effects on hunger, food intake, satiety and skin temperature in humans Endocrine connections 2016;5 (2):65-73 [PubMed]
  • Hollak Carla E. M., Biegstraaten Marieke, Baumgartner Matthias R., Belmatoug Nadia, Bembi Bruno, Bosch Annet, Brouwers Martijn, Dekker Hanka, Dobbelaere Dries, Engelen Marc, Groenendijk Marike C., Lachmann Robin, Langendonk Janneke G., Langeveld Mirjam, Linthorst Gabor, Morava Eva, Poll-The Bwee Tien, Rahman Shamima, Rubio-Gozalbo M. Estela, Spiekerkoetter Ute, Treacy Eileen, Wanders Ronald, Zschocke Johannes, Hagendijk Rob Position statement on the role of healthcare professionals, patient organizations and industry in European Reference Networks Orphanet journal of rare diseases 2016;11 (1):7 [PubMed]
  • Rutten M, Ciet P, van den Biggelaar R, Oussoren E, Langendonk J G, van der Ploeg A T, Langeveld M Severe tracheal and bronchial collapse in adults with type II mucopolysaccharidosis Orphanet journal of rare diseases 2016;11:50 [PubMed]

2015

  • Langeveld Mirjam, DeVries J. Hans The long-term effect of energy restricted diets for treating obesity Obesity (Silver Spring, Md.) 2015;23 (8):1529-1538 [PubMed]

2013

  • Langeveld Mirjam, de Vries J. H. Hans Het magere resultaat van diëten Nederlands tijdschrift voor geneeskunde 2013;157 (29):A6017 [PubMed]

2012

  • Langeveld Mirjam, van den Berg Sjoerd A. A., Bijl Nora, Bijland Silvia, van Roomen Cindy P., Houben-Weerts Judith H., Ottenhoff Roelof, Houten Sander M., van Dijk Ko Willems, Romijn Johannes A., Groen Albert K., Aerts Johannes M., Voshol Peter J. Treatment of genetically obese mice with the iminosugar N-(5-adamantane-1-yl-methoxy-pentyl)-deoxynojirimycin reduces body weight by decreasing food intake and increasing fat oxidation Metabolism: clinical and experimental 2012;61 (1):99-107 [PubMed]

2011

  • Biegstraaten M., van Schaik I. N., Aerts J. M. F. G., Langeveld M., Mannens M. M. A. M., Bour L. J., Sidransky E., Tayebi N., Fitzgibbon E., Hollak C. E. M. A monozygotic twin pair with highly discordant Gaucher phenotypes Blood cells, molecules & diseases 2011;46 (1):39-41 [PubMed]
  • Aerts Johannes M., Boot Rolf G., van Eijk Marco, Groener Johanna, Bijl Nora, Lombardo Elisa, Bietrix Florence M., Dekker Nick, Groen Albert K., Ottenhoff Roelof, van Roomen Cindy, Aten Jan, Serlie Mireille, Langeveld Mirjam, Wennekes Tom, Overkleeft Hermen S. Glycosphingolipids and insulin resistance Advances in experimental medicine and biology 2011;721:99-119 [PubMed]

2010

  • Aerts J. M., Boot R. G., van Eijk M., Groener J., Bijl N., Lombardo E., Bietrix F. M., Dekker N. [=Nick], Groen A. K., Ottenhoff R., van Roomen C., Aten J., Serlie M., Langeveld M., Wennekes T., Overkleeft H. S. Glycosphingolipids and insulin resistancein: A. Cowart, editors. Sphingolipids and Metabolic Disease. S.l.: Landes Bioscience and Springer Science; 2010. p. 20-41, ISBN 9781461406501

2009

  • Langeveld Mirjam, Aerts Johannes M. F. G. Glycosphingolipids and insulin resistance Progress in lipid research 2009;48 (3-4):196-205 [PubMed]
  • de Fost M., Langeveld M., Franssen R., Hutten B. A., Groener J. E. M., de Groot E., Mannens M. M., Bikker H., Aerts J. M. F. G., Kastelein J. J. P., Hollak C. E. M. Low HDL cholesterol levels in type I Gaucher disease do not lead to an increased risk of cardiovascular disease Atherosclerosis 2009;204 (1):267-272 [PubMed]
  • Bijl Nora, Sokolović Milka, Vrins Carlos, Langeveld Mirjam, Moerland Perry D., Ottenhoff Roelof, van Roomen Cindy P. A. A., Claessen Nike, Boot Rolf G., Aten Jan, Groen Albert K., Aerts Johannes M. F. G., van Eijk Marco Modulation of Glycosphingolipid Metabolism Significantly Improves Hepatic Insulin Sensitivity and Reverses Hepatic Steatosis in Mice Hepatology (Baltimore, Md.) 2009;50 (5):1431-1441 [PubMed]
  • Langeveld Mirjam Studies on the role of glycosphingolipids in metabolism 2009. 204p. [UvA Dissertations Online] University of Amsterdam. (Supervisors: Aerts J. M. F. G., Hollak C. E. M.)

2008

  • Langeveld M., de Fost M., Aerts J. M. F. G., Sauerwein H. P., Hollak C. E. M. Overweight, insulin resistance and type II diabetes in type I Gaucher disease patients in relation to enzyme replacement therapy Blood cells, molecules & diseases 2008;40 (3):428-432 [PubMed]
  • Ghauharali-van der Vlugt Karen, Langeveld Mirjam, Poppema Aldi, Kuiper Sijmen, Hollak Carla E. M., Aerts Johannes M., Groener Johanna E. M. Prominent increase in plasma ganglioside GM3 is associated with clinical manifestations of type I Gaucher disease Clinica chimica acta; international journal of clinical chemistry 2008;389 (1-2):109-113 [PubMed]
  • Langeveld Mirjam, Ghauharali Karen J. M., Sauerwein Hans P., Ackermans Mariette T., Groener Johanna E. M., Hollak Carla E. M., Aerts Johannes M., Serlie Mireille J. Type I Gaucher disease, a glycosphingolipid storage disorder, is associated with insulin resistance Journal of clinical endocrinology and metabolism 2008;93 (3):845-851 [PubMed]

2007

  • Langeveld M., Endert E., Wiersinga W. M., Aerts J. M. F. G., Hollak C. E. M. Hypermetabolism in Gaucher disease type I is not associated with altered thyroid hormone levels Journal of inherited metabolic disease 2007;30 (6):985 [PubMed]
  • Langeveld Mirjam, Scheij Saskia, Dubbelhuis Peter, Hollak Carla E. M., Sauerwein Hans P., Simons Peter, Aerts Johannes M. F. G. Very low serum adiponectin levels in patients with type 1 Gaucher disease without overt hyperglycemia Metabolism: clinical and experimental 2007;56 (3):314-319 [PubMed]

2006

  • Boot Rolf G., Verhoek Marri, Langeveld Mirjam, Renkema G. Herma, Hollak Carla E. M., Weening Jan J., Donker-Koopman Wilma E., Groener Johanna E., Aerts Johannes M. F. G. CCL18: a urinary marker of Gaucher cell burden in Gaucher patients Journal of inherited metabolic disease 2006;29 (4):564-571 [PubMed]
  • Langeveld M., Gamadia L. E., ten Berge I. J. M. T-lymphocyte subset distribution in human spleen European journal of clinical investigation 2006;36 (4):250-256 [PubMed]

2005

  • Aerts J. M. F. G., van Breemen M. J., Bussink A. P., Brinkman J., Hollak C. E. M., Langeveld M., Linthorst G. E., Vedder A. C., de Fost M. The blood-brain barrier and treatment of lysosomal storage disease INT CONGRESS SERIES 2005;1277:19-31
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