Funding transnational collaborative research through joint transnational calls is one of the major objectives of E-Rare. This is the most important and effective joint activity to enhance the cooperation between European scientists working on rare diseases and thus reducing the fragmentation of research in this field. E-Rare launches calls on a yearly basis. The topic and eligibility criteria are specified every year and therefore may vary from one call to the other.
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Arne Hansen | Department of Experimental Pharmacology and Toxicology University Medical Center Hamburg-Eppendorf Hamburg | Hamburg, Germany |
Arnaud Klein | Department of Muscle Therapy Institute of Myology Paris | Paris, France |
Beatriz Llamusi | Department of Genetic Fundacion Investigacion Clinico de Valencia Valencia | Valencia, Spain |
Myotonic Dystrophies (Steinert diseases) are the most common muscular dystrophy in adults. Patients suffer from multiple symptoms among which cardiac conduction defects and arrhythmias. Importantly, the mechanisms leading to such fatal cardiac dysfunctions are still unknown. We propose to create new physiological models, including, fly models, mouse models and engineered 3D heart tissue derived from human iPS cells, to study the cardiac alterations of Myotonic Dystrophies. Next, we will take advantage of these models to identify pharmacological compounds able to restore normal cardiac function. A critical issue as there is no available therapy for Myotonic Dystrophic patients.
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For more information ABOUT E-Rare please contact:
E-Rare Coordinator
Daria JULKOWSKA
Tel.: +33(0) 1 78 09 80 78
+33(0) 6 20 14 13 81
daria.julkowska@agencerecherche.fr
Agence Nationale de la Recherche - ANR
Health & Biology Department
50 Avenue Daumesnil
75012 Paris, France