CORDIS Project
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This project aims to develop PTI5803, a first-in-class drug targeting the Pannexin 1 channel to treat drug-resistant epilepsy in children. By effectively inhibiting seizures while preserving cognitive functions, this innovative treatment seeks to address significant unmet medical needs in pediatric epilepsy.
Epilepsy is a group of syndromes with high unmet medical need, especially in children as frequent seizures affect the immature brain and its development.
In some orphan syndromes, drug resistance is 70% with dramatic consequences for patients and their families.
Novel treatments must prevent the negative effects of seizures, but also minimize off-target effects on brain development and cognitive functions.
Our solution is the specific blockade of the activated form of the Pannexin 1 (Panx1) chan…
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