Lissencephaly therapeutic agent

摘要

An object of the present invention is to provide a medicament and method for treating lissencephaly patients. The present invention provides a lissencephaly therapeutic or preventive agent comprising a compound represented by the general formula (I):;
wherein R1 is lower alkyl substituted with lower alkoxy, lower alkyl substituted with a heterocyclic group, a heterocyclic group, or a group represented by the formula (IIa):
;
wherein R4 is lower alkyl, R3 is lower alkylene, and m is an integer of 1 to 6;
R2 is lower alkyl optionally substituted with phenyl; andR3 is lower alkyl optionally substituted with halogen, lower alkoxy, or phenyl; condensed polycyclic hydrocarbon; or hydrogen.

主权项

1. A method for treating lissencephaly comprising administering to a lissencephaly patient or a pregnant woman who is carrying a lissencephaly fetus, a compound represented by the general formula (I): wherein R1 is a linear or branched C1-6 alkyl substituted with C1-6 alkoxy; a linear or branched C1-6 alkyl substituted with a heterocyclic group; a heterocyclic group, or a group represented by the formula (IIa): wherein R4 is a linear or branched C1-6 alkyl; R5 is C1-4 alkylene, and m is an integer of 1 to 6; R2 is a linear or branched C1-6 alkyl optionally substituted with phenyl; and R3 is a linear or branched C1-6 alkyl optionally substituted with halogen, a C1-6 alkoxy, or phenyl; a C3-6 cycloalkyl optionally substituted with halogen, C1-6 alkoxy, or phenyl; a condensed polycyclic hydrocarbon; or hydrogen.