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Addressing disease heterogeneity in myotonic dystrophy. Towards personalized intervention.
This seminar will focus on the development of individualized interventions in myotonic dystrophy that take into account the clinical heterogeneity between patients and that meet personalized treatment goals.
The topic will be addressed from the the patient perspective (interview by Alain Geille, president Euro-DyMA), the perspective of a clinician and clinical researcher (Prof. Baziel van Engelen, Neurology, Radboudumc) and a basic researcher (Prof. Peter-Bram 't Hoen, Bioinformatics, Radboudumc). Experiences and lessons from the OPTIMISTIC trial with Cognitive Behavioural Therapy as a personalized intervention will be shared. Molecular biomarker signatures associated with heterogeneity in clinical phenotypes and how this motivates development of drug repurposing strategies in the ReCognitION project will be presented.

Jun 10, 2021 04:00 PM in Paris

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