ANR HypoGluTx

This project aims to develop new treatments for rare neurological disorders, such as certain forms of epilepsy, autism, and intellectual disability, by repurposing existing drugs. These disorders are caused by genetic mutations, including mutations in the SHANK3 gene, which disrupt communication between brain cells, particularly at synapses, which are essential for learning and behavior.

Although these disorders are distinct, they share common biological dysregulations at glutamatergic synapses. By studying patient-derived cells and animal models, the project seeks to gain a better understanding of these mechanisms. Its primary objective is to identify and repurpose drugs capable of restoring normal brain function. This approach could accelerate access to effective treatments and improve the quality of life of patients and their families.