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Charlotte Höybye Department of Endocrinology, Department of Molecular Medicine and Surgery, Metabolism and Diabetology, Karolinska University Hospital, 171 76 Stockholm, Sweden
Department of Endocrinology, Department of Molecular Medicine and Surgery, Metabolism and Diabetology, Karolinska University Hospital, 171 76 Stockholm, Sweden

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Erik Wahlström Department of Endocrinology, Department of Molecular Medicine and Surgery, Metabolism and Diabetology, Karolinska University Hospital, 171 76 Stockholm, Sweden

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Petra Tollet-Egnell Department of Endocrinology, Department of Molecular Medicine and Surgery, Metabolism and Diabetology, Karolinska University Hospital, 171 76 Stockholm, Sweden

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Gunnar Norstedt Department of Endocrinology, Department of Molecular Medicine and Surgery, Metabolism and Diabetology, Karolinska University Hospital, 171 76 Stockholm, Sweden

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with healthy controls, and changes in metabolic profile during GH treatment and after discontinuation of GH replacement was observed. The aims of this study were to detect the levels of serum metabolome in GHD patients and healthy subjects and to find

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A C Paepegaey Nutrition Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, French Reference Center for Prader-Willi Syndrome, Sorbonne Université, Paris, France

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M Coupaye Nutrition Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, French Reference Center for Prader-Willi Syndrome, Sorbonne Université, Paris, France

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A Jaziri Nutrition Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, French Reference Center for Prader-Willi Syndrome, Sorbonne Université, Paris, France

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F Ménesguen Nutrition Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, French Reference Center for Prader-Willi Syndrome, Sorbonne Université, Paris, France

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B Dubern Nutrition and Gastroenterology Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Armand Trousseau Children’s Hospital, Paris, France

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M Polak Pediatric Endocrinology, Diabetology and Gynecology Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Necker Enfants Malades Hospital University Hospital, Paris, France

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J M Oppert Nutrition Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, French Reference Center for Prader-Willi Syndrome, Sorbonne Université, Paris, France

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M Tauber Pediatric Endocrinology Department, Children’s Hospital, French Reference Center for Prader-Willi Syndrome, Toulouse, France

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G Pinto Pediatric Endocrinology, Diabetology and Gynecology Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Necker Enfants Malades Hospital University Hospital, Paris, France

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C Poitou Nutrition Department, Assistance-Publique Hôpitaux de Paris (AP-HP), Pitié-Salpêtrière Hospital, French Reference Center for Prader-Willi Syndrome, Sorbonne Université, Paris, France
INSERM, UMRS 1166, Nutriomic Group 6, Paris, France
Sorbonne Université, UMRS1166, Paris, France

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, sex-hormone treatment in adulthood, GH treatment during childhood and/or in adulthood. The two-sided significance level was fixed at 5%. Statistical analyses were performed using JMP statistic software (SAS Institute Inc. Cary, NC, USA

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Ekaterina Koledova Biopharma Global Medical Affairs, General Medicine and Endocrinology, Merck KGaA, Darmstadt, Germany

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George Stoyanov Biopharma Global Medical Affairs, General Medicine and Endocrinology, Merck KGaA, Darmstadt, Germany

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Leroy Ovbude Business & Decision Life Sciences, Brussels, Woluwe-Saint-Lambert, Belgium

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Peter S W Davies Children’s Nutrition Research Centre, Faculty of Medicine, University of Queensland, Brisbane, Queensland, Australia

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catch-up growth ( 1 , 2 , 3 ). The aim of GH treatment is to initiate catch-up growth and improve adult height and metabolic parameters, which requires long-term commitment from the patient and/or carer for regular injections. The length of continuous

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Lukas Plachy Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Lenka Petruzelkova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Petra Dusatkova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Klara Maratova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Dana Zemkova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Lenka Elblova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Vit Neuman Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Stanislava Kolouskova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Barbora Obermannova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Marta Snajderova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Zdenek Sumnik Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Jan Lebl Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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Stepanka Pruhova Department of Pediatrics, 2nd Faculty of Medicine, Charles University in Prague and University Hospital Motol, Prague, Czech Republic

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between children with monogenic FSS compared to those with no monogenic FSS etiology elucidated. Monogenic FSS No monogenic etiology elucidated P -value Height before GH treatment (SD) −3.1 (−3.6 to −2.7) −3.0 (−3.5 to −2

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Imane Benabbad Endocrinology and Diabetes Unit, Eli Lilly, Neuilly-sur-Seine, France

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Myriam Rosilio Endocrinology and Diabetes Unit, Eli Lilly, Neuilly-sur-Seine, France

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Maité Tauber Department Endocrine, Bone Diseases, Genetics, Obesity, and Gynecology Unit, Children’s Hospital, University Hospital, Toulouse, France

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Emmanuel Paris BioClinica, Lyon, France

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Anne Paulsen Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Universitaire Robert-Debré, Department of Pediatric Endocrinology and Diabetology, and Centre de Référence des Maladies Endocriniennes Rares de la Croissance, Paris, France

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Lovisa Berggren Eli Lilly and Company, Bad Homburg, Germany

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Hiren Patel Eli Lilly and Company, Indianapolis, Indiana, USA

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Jean-Claude Carel Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Universitaire Robert-Debré, Department of Pediatric Endocrinology and Diabetology, and Centre de Référence des Maladies Endocriniennes Rares de la Croissance, Paris, France
Assistance Publique-Hôpitaux de Paris (AP-HP), Hôpital Universitaire Robert-Debré, Department of Pediatric Endocrinology and Diabetology, and Centre de Référence des Maladies Endocriniennes Rares de la Croissance, Paris, France

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the Phoenix Study Group
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cause short stature have been excluded ( 1 ). Various studies have shown that growth hormone (GH) treatment of children with ISS can increase their growth velocity and, to a lesser extent, adult height ( 1 , 2 , 3 , 4 , 5 , 6 ). The effect on height

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Régis Coutant Department of Pediatric Endocrinology and Diabetology, Reference Center for Rare Pituiatry Diseases, University Hospital of Angers, Angers, France

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Maithé Tauber Reference Center for the Prader-Willi syndrome and other rare obesities with feeding disorders (PRADORT), Children Hospital, CHU Toulouse, Toulouse, France
Pediatric team of the Clinical Investigation Center 9302/INSERM, Hospital of Children, Toulouse, France
Institut Toulousain des Maladies Infectieuses et Inflammatoires (Infinity), INSERM UMR1291 - CNRS UMR5051 - Université Toulouse III, Toulouse, France

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Béatrice Demaret GRANDIR - French Growth Disorders Association, Asnières-sur-Seine, France

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Robin Henocque Pfizer France, Paris France

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Yves Brault Pfizer France, Paris France

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François Montestruc eXYSTAT, Malakoff, France

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Olivier Chassany Health Economics Clinical Trial Unit (URC-ECO), Hospital of Hotel-Dieu, AP-HP, Paris, France
Patient-Reported Outcomes Unit (PROQOL), UMR 1123, University Paris Cité, INSERM, Paris, France

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Michel Polak Hôpital Universitaire Necker Enfants Malades, Pediatric Endocrinology, Gynecology and Diabetology, Imagine Institute, INSERM U1163, Cochin Institute, INSERM U1016, Centre de référence des pathologies endocriniennes rares de la croissance et du développement, Université de Paris Cité, Paris, France

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the QOLITHOR Study Group
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the QOLITHOR Study Group

ρ = 0 ( α  = 0.05 two-sided) for the level of the overall life interference total score according to the GH treatment duration (analyzed as a continuous variable) had 90% power to detect a correlation coefficient ρ of at least 0.25. The

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Thomas Reinehr Pediatric Endocrinology, Diabetes and Nutrition Medicine, Vestische Children’s Hospital, University of Witten/Herdecke, Datteln, Germany

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Martin Carlsson Endocrine Care, Pfizer Inc, New York, New York, USA

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Dionisios Chrysis Division of Pediatric Endocrinology, University of Patras, Patras, Greece

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Cecilia Camacho-Hübner Endocrine Care, Pfizer Inc, New York, New York, USA

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of GH treatment and adult height in children with GHD is lower compared to target height even though the children were treated adequately with growth hormone (GH) ( 13 ), we hypothesize that adult height prediction based on bone age will overestimate

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Gudmundur Johannsson Department of Internal Medicine and Clinical Nutrition, Sahlgrenska Academy, University of Gothenburg, Gothenburg, Sweden

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Martin Bidlingmaier Medizinische Klinik und Poliklinik IV, Klinikum der Universität München, Munich, Germany

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Beverly M K Biller Neuroendocrine Unit, Massachusetts General Hospital, Boston, Massachusetts, USA

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Margaret Boguszewski Federal University of Parana, Curitiba, Brazil

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Felipe F Casanueva Department of Medicine, Complejo Hospitalario Universitario de Santiago, Santiago de Compostela, Spain

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Philippe Chanson Assistance Publique-Hôpitaux de Paris, and Inserm, Paris, France

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Peter E Clayton Developmental Biology & Medicine, Faculty of Biology, Medicine & Health, University of Manchester, Manchester, UK

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Catherine S Choong Department of Endocrinology, Princess Margaret Hospital & School of Medicine, University of Western Australia, Western Australia, Australia

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David Clemmons Department of Medicine, University of North Carolina, Chapel Hill, North Carolina, USA

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Mehul Dattani Great Ormond Street Institute of Child Health, London, UK

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Jan Frystyk Department of Endocrinology, Odense University Hospital, Odense, Denmark

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Ken Ho Princess Alexandra Hospital and University of Queensland, Brisbane, Australia

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Andrew R Hoffman Department of Medicine, Stanford University and VA Palo Health Care System, Palo Alto, California, USA

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Reiko Horikawa National Center for Child Health and Development, Tokyo, Japan

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Anders Juul Department of Growth and Reproduction, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark

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John J Kopchick Edison Biotechnology Institute and Heritage College of Osteopathic Medicine, Ohio University, Athens, Ohio, USA

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Xiaoping Luo Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China

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Sebastian Neggers Section of Endocrinology, Department of Medicine, Pituitary Centre Rotterdam, Erasmus University Medical Centre, Rotterdam, the Netherlands

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Irene Netchine Service d’Explorations Fonctionnelles Endocriniennes, AP-HP, Hôpital Trousseau, Sorbonne Université, INSERM UMRs 938, Paris, France

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Daniel S Olsson Department of Endocrinology, Institute of Medicine, Sahlgrenska Academy, University of Gothenburg and Sahlgrenska University Hospital, Gothenburg, Sweden

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Sally Radovick Rutgers University-Robert Wood Johnson Medical School, New Brunswick, New Jersey, USA

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Ron Rosenfeld Department of Pediatrics, Oregon Health Science University, Portland, Oregon, USA

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Richard J Ross University of Sheffield, Sheffield, UK

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Katharina Schilbach Medizinische Klinik und Poliklinik IV, Klinikum der Universität München, Munich, Germany

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Paulo Solberg Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brazil

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Christian Strasburger Charité-Universitätsmedizin, Berlin, Germany

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Peter Trainer The Christie NHS Foundation Trust, University of Manchester, Manchester, UK

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Kevin C J Yuen Barrow Pituitary Center, Barrow Neurological Institute, Department of Neuroendocrinology, University of Arizona College of Medicine, Phoenix, Arizona, USA

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Kerstin Wickstrom Medical Products Agency, Uppsala, Sweden

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Jens O L Jorgensen Aarhus University Hospital, Aarhus, Denmark

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on behalf of the Growth Hormone Research Society
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endpoint in trials involving GH treatment and medical treatment of acromegaly, neither GH nor IGF-I has been subject to a structured evaluation as biomarkers, nor do we have a comprehensive definition of clinical endpoints for the treatment of GH

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Alicia Romano Department of Pediatrics, New York Medical College, Valhalla, New York, USA

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Juan Pablo Kaski Centre for Inherited Cardiovascular Diseases, Great Ormond Street Hospital & UCL Institute of Cardiovascular Science, London, UK

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Jovanna Dahlgren Department of Paediatrics, Region Västra Götaland, Sahlgrenska University Hospital, Gothenburg, Sweden

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Nicky Kelepouris US Medical Affairs, Novo Nordisk Inc., Plainsboro, New Jersey, USA

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Alberto Pietropoli Global Medical Affairs, Novo Nordisk Health Care AG, Zurich, Switzerland

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Tilman R Rohrer Department of Pediatric Endocrinology, University Children’s Hospital, Saarland University Medical Center, Homburg, Germany

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Michel Polak Paediatric Endocrinology, Diabetology and Gynaecology Department, Hôpital Universitaire Necker Enfants-Malades, AP-HP, Université de Paris, Imagine Institute, Paris, France

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stenosis (PVS; prevalence 50–60%), hypertrophic cardiomyopathy (HCM; 20%) and atrial septal defects (6–10%) ( 1 ). Growth hormone (GH) treatment in patients with NS improves height velocity, height standard deviation score (SDS) and adult height, with a

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Charlotte Höybye Patient Area Endocrinology and Nephrology, Infection and Inflammation Theme, Karolinska University Hospital, Stockholm, Sweden
Department of Molecular Medicine and Surgery, Karolinska Institute, Stockholm, Sweden

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Laia Faseh Department of Molecular Medicine and Surgery, Karolinska Institute, Stockholm, Sweden

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Christos Himonakos Department of Molecular Medicine and Surgery, Karolinska Institute, Stockholm, Sweden
Department of Medicine, Karlstad Hospital, Karlstad, Sweden

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Tomasz Pielak NUTOPI Sp. z o. o., Poznan, Poland

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Jesper Eugen-Olsen Clinical Research Centre, Copenhagen University Hospital Hvidovre, Hvidovre, Denmark

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shown that GH treatment improves the above-mentioned risk factors for CVD ( 1 , 2 , 3 , 5 , 6 , 7 , 12 , 13 , 14 ), but it is currently unknown if GHD and long-term GHD treatment influences the level of low-grade inflammation. Urokinase

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