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Sanfilippo disease (mucopolysaccharidosis type III): Early diagnosis and treatment de Ruijter, J.

Publication date 2013

Link to publication

Citation for published version (APA): de Ruijter, J. (2013). Sanfilippo disease (mucopolysaccharidosis type III): Early diagnosis and treatment.

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Download date:29 Sep 2021 SANFILIPPO DISEASE (MUCOPOLYSACCHARIDOSIS TYPE III): EARLY DIAGNOSIS AND TREATMENT

Jessica de Ruijter ISBN: 978-94-6182-301-4 Printing of this thesis was made possible by donations from Synageva BioPharma Corp., BioMarin Pharmaceutical Inc., Shire Human Genetic Therapies, Actelion Pharmaceuticals, and Genzyme Nederland. Layout and Printing: Off Page, www.offpage.nl Cover Illustration: Le Passe-Muraille, a statue in Montmartre, Paris. The cover represents overcoming the blood-brain-barrier (BBB), one of the main hurdles to overcome, and the key for successful therapy of treating brain pathology in MPS III. Copyright © 2013 by J. de Ruijter. All rights reserved. No part of this book may be reproduced, stored in a retrieval system, or transmitted in any form of by any mean, without prior permission of the author. SANFILIPPO DISEASE (MUCOPOLYSACCHARIDOSIS TYPE III): EARLY DIAGNOSIS AND TREATMENT

ACADEMISCH PROEFSCHRIFT

ter verkrijging van de graad van doctor aan de Universiteit van Amsterdam op gezag van de Rector Magnificus prof. dr. D.C. van den Boom ten overstaan van een door het college voor promoties ingestelde commissie, in het openbaar te verdedigen in de Agnietenkapel op woensdag 18 september 2013, te 14:00 uur

door

Jessica de Ruijter geboren te ’s Gravenhage PROMOTIECOMMISSIE

Promotor: Prof. dr. F.A. Wijburg Overige leden: Prof. dr. D.A.J.P. Denys Prof. dr. R.C.M. Hennekam Prof. dr. C.E.M. Hollak Prof. dr. B.T. Poll-The Dr. G. Visser Prof. dr. R.J.A. Wanders

Faculteit der Geneeskunde