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MCDS-Therapy - Repurposing of carbamazepine for treatment of skeletal dysplasia

3 - Good health and well-being; Research Projects; 2017; 2018; 2019; 2020; 2021; 2022

The MCDS-Therapy team is working to alleviate the pain and bone malformations that patients with the rare genetic skeletal condition MCDS live with on a daily basis. The project aims to develop a low-cost treatment for metaphyseal chondrodysplasia type schmid (MCDS) by repurposing carbamazepine, a drug that is currently marketed to treat a completely different condition. To do this a worldwide clinical trial is planned. Ultimately, the team plans to pioneer an academic drug repurposing pathway to deliver a transformative treatment for a rare patient population from bench to bedside.

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