EndoCompass project: research roadmap for growth disorders

Evelien F. Gevers, Centre for Endocrinology, William Harvey Research Institute, Barts and The London Medical School, Queen Mary University of London, London EC1M 6BQ, United Kingdom.
Anita C. Hokken-Koelega, Department of Paediatrics, Erasmus University Medical Centre Rotterdam, Sophia Children's Hospital, 3015 CN Rotterdam, The Netherlands.
Maithe Tauber, Centre de Référence Maladies Rares PRADORT (syndrome de Prader-Willi et autres Obésités Rares avec Troubles du comportement alimentaire), Hôpital des Enfants, CHU Toulouse, Université Toulouse III, 31300 Toulouse, France.
Gerhard Binder, Pediatric Endocrinology, University Children's Hospital, 72076 Tübingen, Germany.
Elena G. Bochukova, Blizard Institute, Barts and The London Medical School, Queen Mary University of London, London E1 2AT, United Kingdom.
Sebastien G. Bouret, Laboratory of Development and Plasticity of the Neuroendocrine Brain, Lille Neuroscience and Cognition, University of Lille, Inserm, CHU Lille, 59000 Lille, France.
Assumpta Caixàs, Endocrinology and Nutrition Department, Parc Taulí Hospital Universitari, Institut d'Investigació, Innovació Parc Taulí (I3PT-CERCA), 08208 Sabadell, Spain.
Justin H. Davies, Faculty of Medicine, University of Southampton, Southampton SO16 6YD, United Kingdom.
Andrew Dauber, Division of Endocrinology, Children's National Hospital, Washington, DC 20010, United States.
Thomas Edouard, Department of Endocrinology, Bone Diseases and Genetics, Reference Centre for Rare Diseases of Growth and Development, FIRENDO network, Children's Hospital, University Hospital of Toulouse, 31059 Toulouse, France.
Thomas Eggermann, Centre for Human Genetics and Genome Medicine, Medical Faculty, RWTH University Aachen, Aachen 52074, Germany.
Eloise Giabicani, Explorations Fonctionelles Endocriennes, Centre de Recherche Saint Antoine, APHP, Hôpital Armand Trousseau, Sorbonne Universite, F-75012 Paris, France.
Irène Netchine, Explorations Fonctionelles Endocriennes, Centre de Recherche Saint Antoine, APHP, Hôpital Armand Trousseau, Sorbonne Universite, F-75012 Paris, France.
Ola Nilsson, Pediatric Endocrinology Unit and Center for Molecular Medicine, Department of Women's and Children's Health, Karolinska Institute, Stockholm 171 77, Sweden.
Kristina Saravinovska, Clinic of Endocrinology, Diabetes and Metabolic Diseases, University Clinical Centre of Serbia, 11000 Belgrade, Serbia.
Manouk van der Steen, Department of Pediatrics, Division of Endocrinology, Amalia Children's Hospital, Radboud University Medical Center, 6525 GA Nijmegen, The Netherlands.
Marco Tartaglia, Molecular Genetics and Functional Genomics, Bambino Gesù Children's Hospital IRCCS, Rome 00143, Italy.
Katrina Tatton-Brown, South West Thames Regional Genomics Service, St George's University Hospitals NHS Foundation Trust, London SW17 0RE, United Kingdom.
I Karen Temple, Faculty of Medicine, University of Southampton, Southampton SO16 6YD, United Kingdom.
Armelle Yart, RESTORE Research Centre, Université de Toulouse, CNRS, INSERM, EFS, 31100 Toulouse, France.
Martin Zenker, Institute of Human Genetics, University Hospital Magdeburg, Otto von Guericke University Magdeburg, 39120 Magdeburg, Germany.

Document Type

Journal Article

Publication Date

10-17-2025

Journal

European journal of endocrinology

Volume

193

Issue

Supplement_2

DOI

10.1093/ejendo/lvaf070

Keywords

Noonan syndrome; Prader-Willi syndrome; Silver-Russell syndrome; growth hormone; imprinting disorders; short stature; tall stature

Abstract

BACKGROUND: Endocrine science remains underrepresented in European Union research programs despite the fundamental role of hormone health in human wellbeing. Analysis of the CORDIS database reveals a persistent gap between the societal impact of endocrine disorders and their research prioritization. At the national funding level, endocrine societies report limited or little attention of national research funding toward endocrinology. The EndoCompass project-a joint initiative between the European Society of Endocrinology and the European Society of Pediatric Endocrinology, aimed to identify and promote strategic research priorities in endocrine science to address critical hormone-related health challenges. METHODS: Research priorities were established through comprehensive analysis of the EU CORDIS database covering the Horizon 2020 framework period (2014-2020). Expert consultation in growth disorders was conducted to identify key research priorities, followed by broader stakeholder engagement, including society members and patient advocacy groups. RESULTS: Research priorities encompass genetic diagnosis of growth disorders; growth plate-targeted therapies; molecular mechanisms of Silver-Russell syndrome and imprinting disorders; hypothalamic dysfunction in Prader-Willi syndrome; and characterization of Noonan syndrome and tall stature conditions. Emphasis is placed on creating disease registries to facilitate outcome studies and developing precision therapeutics based on growth regulation pathways. CONCLUSIONS: This component of the EndoCompass project provides an evidence-based roadmap for strategic research investment. This framework identifies crucial investigation areas into growth disorder pathophysiology, prevention, and treatment strategies, ultimately aimed at reducing the burden of these disorders on individuals and society. The findings support the broader EndoCompass objective of aligning research funding with areas of highest potential impact in endocrine health.

Department

Pediatrics

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