Master di II Livello in Endocrinologia e Diabetologia Pediatrica Scuola di Pediatria

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1 Advanced Therapies in Pediatric Endocrinology and Diabetology With an unrestricted educational grant from Master di II Livello in Endocrinologia e Diabetologia Pediatrica Scuola di Pediatria

2 Endocrine Development Vol. 30 Series Editor P.-E. Mullis Bern

3 Workshop, Rome, October 27 28, 2014 Advanced Therapies in Pediatric Endocrinology and Diabetology Volume Editors Marco Cappa Rome Stefano Cianfarani Rome Lucia Ghizzoni Turin Sandro Loche Cagliari Mohamad Maghnie Genoa 26 figures, 6 in color and 10 tables, 2016 Basel Freiburg Paris London New York Chennai New Delhi Bangkok Beijing Shanghai Tokyo Kuala Lumpur Singapore Sydney

4 Endocrine Development Founded 1999 by Martin O. Savage, London Marco Cappa Endocrinology and Diabetology in Diabetes Units D.P.U.O. Bambino Gesù Children s Hospital IRCCS Rome, Italy Lucia Ghizzoni Division of Endocrinology, Diabetology and Metabolism Department of Internal Medicine University of Turin Turin, Italy Stefano Cianfarani Molecular Endocrinology Unit D.P.U.O. Bambino Gesù Children s Hospital IRCCS Tor Vergata University Rome, Italy Sandro Loche Regional Hospital for Microcytaemia Cagliari, Italy Mohamad Maghnie Department of Pediatrics IRCCS G. Gaslini University of Genoa Genoa, Italy Library of Congress Cataloging-in-Publication Data Advanced therapies in pediatric endocrinology and diabetology : workshop, Rome, October 27-28, 2014 / volume editors Marco Cappa, Stefano Cianfarani, Lucia Ghizzoni, Sandro Loche, Mohamad Maghnie. p. ; cm. -- (Endocrine development ; vol. 30) Includes bibliographical references and index. ISBN (hard cover : alk. paper) -- ISBN (electronic version) I. Cappa, Marco, editor. II. Cianfarani, Stefano, editor. III. Ghizzoni, Lucia, editor. IV. Loche, Sandro, editor. V. Maghnie, Mohamad, editor. VI. Series: Endocrine development ; v. 30. [DNLM: 1. Endocrine System Diseases--therapy--Congresses. 2. Child--Congresses. W1 EN3635 v / WS 330] RJ dc Bibliographic Indices. This publication is listed in bibliographic services, including Current Contents and PubMed/MEDLINE. Disclaimer. The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publisher and the editor(s). The appearance of advertisements in the book is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements. Drug Dosage. The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any change in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug. All rights reserved. No part of this publication may be translated into other languages, reproduced or utilized in any form or by any means electronic or mechanical, including photocopying, recording, microcopying, or by any information storage and retrieval system, without permission in writing from the publisher. Copyright 2016 by S. Karger AG, P.O. Box, CH 4009 Basel (Switzerland) Printed in Germany on acid-free and non-aging paper (ISO 9706) by Kraft Druck, Ettlingen ISSN e-issn ISBN e-isbn

5 Contents VII Preface Cappa, M.; Cianfarani, S. (Rome); Ghizzoni, L. (Turin); Loche, S. (Cagliari); Maghnie, M. (Genoa) 1 Toward Automation of Insulin Delivery Management Solutions for Type 1 Diabetes Nimri, R. (Petah Tikva); Phillip, M. (Petah Tikva/Tel Aviv) 14 Islet Transplantation in Pediatric Patients: Current Indications and Future Perspectives Bertuzzi, F.; Antonioli, B.; Tosca, M.C.; Galuzzi, M.; Bonomo, M.; Marazzi, M.; Colussi, G. (Milan) 23 Glucagon-Like Peptide-1 Receptor Agonist Treatment for Pediatric Obesity Kelly, A.S. (Minneapolis, Minn.) 29 Docosahexaenoic Acid and Its Role in G-Protein-Coupled Receptor 120 Activation in Children Affected by Nonalcoholic Fatty Liver Disease Della Corte, C.; Mosca, A. (Rome); Ionata, A. (Isernia); Nobili, V. (Rome) 37 Noninvasive Prenatal Diagnosis of Congenital Adrenal Hyperplasia Khattab, A.; Yuen, T.; Sun, L.; Yau, M.; Barhan, A.; Zaidi, M. (New York, N.Y.); Lo, Y.M.D. (Hong Kong, SAR); New, M.I. (New York, N.Y.) 42 Recent Advances in Hydrocortisone Replacement Treatment Mallappa, A. (Bethesda, Md.); Debono, M. (Sheffield) 54 Experience with the Histrelin Implant in Pediatric Patients Eugster, E.A. (Indianapolis, Ind.) 60 Different Medications for Hypogonadotropic Hypogonadism Rastrelli, G.; Vignozzi, L.; Maggi, M. (Florence) 79 Long-Acting Growth Hormone: An Update Saenger, P.H.; Mejia-Corletto, J. (New York, N.Y.) 98 C-Type Natriuretic Peptide Analog as Therapy for Achondroplasia Legeai-Mallet, L. (Paris) 106 Therapeutic Neuroendocrine Agonist and Antagonist Analogs of Hypothalamic Neuropeptides as Modulators of the Hypothalamic-Pituitary-Gonadal Axis Newton, C.L.; Anderson, R.C.; Millar, R.P. (Pretoria/Cape Town) 130 Aromatase Inhibitors in the Treatment of Short Stature Hero, M. (Helsinki) V

6 141 Gene Therapy for Rare Central Nervous System Diseases Comes to Age Aubourg, P. (Le Kremlin-Bicêtre) 147 Novel Therapeutic Targets and Drug Candidates for Modifying Disease Progression in Adrenoleukodystrophy Pujol, A. (Barcelona) 161 Author Index 162 Subject Index VI Contents

7 Preface This volume of Endocrine Development is the 6th volume of a series started in 2007 with the proceedings of an outstanding symposium held in Genoa on congenital endocrinopathies, which was followed by documentations of workshops every other year in different Italian cities (Rome, Cagliari and Turin). The aim of the workshops is to provide an update on advanced therapies of endocrine disorders. The program of this workshop covered several topics organized in thematic sessions and plenary lectures. Outstanding experts discussed a number of issues, including new modalities for treating type 1 diabetes like the artificial pancreas and islet transplantation in pediatric patients, novel therapeutic approaches for obesity and the metabolic syndrome, as well as adrenal and gonadal disorders. Recent advances in growth-promoting therapies, such as the C-type natriuretic peptide analogue to treat achondroplasia as well as oxidative stress blockers and gene therapy for patients with adrenoleukodystrophy, have been discussed. The chapters presented are the results of fruitful exchange of scientific information and discussions of experts in the field and we are really grateful to all the participating speakers and authors for sharing their experience and knowledge with us. We are sure that the quality of this book will be as excellent as the workshop. Marco Cappa, Rome Stefano Cianfarani, Rome Lucia Ghizzoni, Turin Sandro Loche, Cagliari Mohamad Maghnie, Genoa VII

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