tailieunhanh - Pompe disease in children and adults: natural course, disease severity and impact on daily life

The transition from efficacy of interventions to effectiveness of delivery strategies is where we so often lose our way. If efficacy is “proven” by techniques such as the randomized controlled trial that screen out the noise of confounding variables, then, ultimately, the techniques to assess effectiveness of delivery strategies and to decide priorities for health sector policy must do just the opposite. They must take into account, they must even grow out of, precisely the messy, contradictory, dissonant noises of real life. In this sense “delivery strategy” is a misleading term,. | ompe disease in children and adults natural course disease severity and impact on daily life Results from an international patient survey Pompe disease in children and adults natural course disease severity and impact on daily life Results from an international patient survey Marloes Hagemans The studies described in this thesis were performed at Erasmus MC University Medical Center Rotterdam the Netherlands and were financially supported by the Princess Beatrix Fund the International Pompe Association and Genzyme Corp. Boston MA. The printing of this thesis was sponsored by Genzyme Europe . and the International Pompe Association. ISBN 90-9020644-2 . Hagemans 2006 All rights reserved. No part of this thesis may be reproduced stored in a retrieval system or transmitted in any form or by any means without the prior written permission of the author. The copyright of the publications remains with the publishers. Layout Tom de Vries Lentsch Cover photography Peter Nicolai Cover design Lennart Nicolai Tom de Vries Lentsch Printed by PrintPartners Ipskamp .