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Financement de l’UE (8 048 715 €) : Réseau européen pour les RASopathies neurodéveloppementales Hor01/06/2023 Programme de recherche et d'innovation de l'UE « Horizon »
Texte
Réseau européen pour les RASopathies neurodéveloppementales
Cardio-facio-cutaneous syndrome, Costello syndrome and SYNGAP1-related encephalopathy are rare neurodevelopmental RASopathies manifesting with severe intellectual disability, seizures and autism spectrum disorder. These symptoms are underdiagnosed and lack approved therapeutic options. EURAS aims to develop new effective therapies and operative disease management for RASopathies. With strong support of patient organizations from 13 countries, a new patient report-based registry for stratification of patient cohorts and evaluation of treatments will be implemented. To clarify the pathophysiology of RASopathies and to identify cellular/molecular “disease signatures” as biomarkers for drug screening and for therapy validation, EURAS will generate new and employ existing disease models. Phenotypical screenings of drug libraries assisted by automated high-resolution, high-content pipelines will be conducted on differentiated, electrically active patient iPSC-derived neurons. Preclinical proof-of-concept trials with repurposed drugs, antisense oligonucleotides and motor-cognitive training will be performed to achieve reversal of seizures and autistic behavioural traits in animal models. To provide patients with less invasive treatments, new technologies for efficient drug delivery of treatments to the brain will be developed and validated. Finally, EURAS establishes Europe-wide patient association to improve awareness and exchange between affected families and to foster their access to knowledge and therapies. With all these measures, EURAS delivers new effective therapies and set the frame for early and accurate diagnosis of neurological symptoms in RASopathies, which are not available at present. Moreover, some results of EURAS will be widely transferable, fostering development of therapies even for unrelated diseases. Thus, EURAS will sustainably improve the lives of affected people and strengthen expertise of health systems in Europe and worldwide.
| The University of Edinburgh | ? |
| AIT Austrian Institute OF Technology GmbH | 429 550 € |
| Centre National de la Recherche Scientifique CNRS | 645 940 € |
| ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM | 552 680 € |
| EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GmbH | 500 000 € |
| Ist-ID Associação do Instituto Superior Tecnico para A Investigacao e O Desenvolvimento | 484 576 € |
| MAX-PLANCK-GESELLSCHAFT ZUR FORDERUNG DER WISSENSCHAFTEN e. V. | 0,00 € |
| NEUWAY PHARMA GmbH | 88 753 € |
| OTTO-VON-GUERICKE-UNIVERSITAET MAGDEBURG | 296 208 € |
| Paracelsus Medizinische Privatuniversitat Salzburg - Privatstiftung | 1 102 938 € |
| RUPRECHT-KARLS-UNIVERSITAET HEIDELBERG | 482 750 € |
| SYNGAP ELTERNHILFE e. V. | 50 000 € |
| TURUN YLIOPISTO | 707 998 € |
| Universidad de Malaga | 376 593 € |
| UNIVERSITAET ULM | 412 751 € |
| UNIVERSITATSKLINIKUM ERLANGEN | 1 292 980 € |
| ZENTRALINSTITUT FUER SEELISCHE GESUNDHEIT | 625 000 € |
https://cordis.europa.eu/project/id/101080580
Cette annonce se réfère à une date antérieure et ne reflète pas nécessairement l’état actuel. L’état actuel est présenté à la page suivante : The University of Edinburgh, Edinburgh, Royaume Uni.
