Ganaxolone for use in treating genetic epileptic disorders
AssigneeMarinus Pharmaceuticals
InventorsLorianne K. Masuoka, Jaakko Lappalainen
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Abstract
The disclosure provides a method of treating a mammal having a genetic epileptic disorder, comprising chronically administering a pharmaceutically acceptable pregnenolone neurosteroid to a mammal having a genetic epileptic disorder in an amount effective to reduce the seizure frequency in the mammal. In certain preferred embodiments, the mammal is a human patient who has a CDKL5 genetic mutation. In certain preferred embodiments, the patient has a low endogenous level of a neurosteroid(s). In certain preferred embodiments, the pregnenolone neurosteroid is ganaxolone.
Claims20
A method of treating a mammal having an epileptic disorder, comprising
- determining whether a mammal has a low level of an endogeneous neurosteroid, and
- if the mammal has the low level of the endogenous neurosteroid, chronically administering a pharmaceutically acceptable pregnenolone neurosteroid to the mammal.
A method of treating an epileptic encephalopathy, comprising
- identifying a human patient suffering from the epileptic encephalopathy,
- determining if the human patient has a low level of an endogenous neurosteroid, and
- if the human patient has a low level of an endogenous neurosteroid, administering the human patient a dosage regimen of a pharmaceutically acceptable pregnenolone neurosteroid in an amount effective to reduce the frequency of seizures in the human patient.
Dependencies are inferred from the claim text.