TY - PAT
T1 - Compounds for treatment of intractable epilepsy and doors syndrome
AU - Verstreken, Patrick
AU - Lüthy, Kevin
AU - Versées, Wim
AU - Fischer, Baptiste
AU - Paesmans, Jone
PY - 2019
Y1 - 2019
N2 - 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.
AB - 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.
UR - https://worldwide.espacenet.com/patent/search/family/057119896/publication/EP3503880A1?q=EP3503880
M3 - Patent
M1 - EP3503880
ER -