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.
COMPOUNDS FOR TREATMENT OF INTRACTABLE EPILEPSY AND DOORS SYNDROME