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Patent

Compounds for treatment of intractable epilepsy and doors syndrome

The present invention relates to the field of neurodegenerative diseases, and the prevention and/or treatment of TBC1D24-associated disorders, such as DOORS syndrome, (intractable) epilepsy, and nonsyndromic deafness. In particular, the present invention relates to phosphoinositide phosphatase inhibitors and screening methods for producing such inhibitors, more particularly, Synaptojanin-1 5' phosphatase inhibitors, to increase phosphatidylinositol 4,5-bisphosphate (PI(4,5)P2) levels in neuronal cells.
Patent Publication Number: EP3503880
Year filing: 2017
Year approval: 2020
Validated for IOF-key: Yes
Attributed to: Associatie KULeuven, Universitaire Associatie Brussel