28 April 2021

Clara van Karnebeek, theme Metabolic diseases, has received a European grant of 1.88 million Euros for the CHARLIE consortium: an international collaboration to use genetic and stem cell technology to develop new treatments for the neurometabolic diseases  Glutaric aciduria type I (GA1) and Pyridoxine-dependent epilepsy (PDE).

The CHARLIE project stands for CHAnging Rare Disorders of LysInE metabolism, a United for Metabolic Diseases (UMD) collaboration with Karlien Coene, theme Metabolic diseases at the Translational Metabolic Laboratory (TML), Hanka Dekker (patient association VKS), and partners from five different countries. The goal is to develop and apply innovative genetic therapy or drugs for these diseases, with an active role for patients. The project is funded by the European Union's Horizon 2020 research and innovation program, under the European Joint Program on Rare Diseases.

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