Treatment of neurological disorders
AssigneePraxis Precision Medicines, Inc.
InventorsMichael Kristopher Mathieu Kahlig, Marion Wittmann, Zoe A. Hughes, Bernard Ravina
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Abstract
The present disclosure is generally directed to methods of treating a disease, disorder, or condition, e.g., a neurological disorder, a disorder associated with excessive neuronal excitability, or a disorder associated with de novo gain-of-function or loss-of-function mutations in major central nervous system sodium channel genes, such as for example, SCN1A, SCN2A, and SCN8A, using a compound of Formula (I) as defined herein or a pharmaceutically acceptable salt thereof.
Claims22
A method of treating a condition relating to aberrant function of a sodium ion channel in a subject in need thereof, said method comprising administering to said subject Compound 1 of the following formula:
- or a pharmaceutically acceptable salt thereof, at a dose of about 0.1 mg/kg to about 10 mg/kg, administered once daily,
- wherein the condition is a genetic epilepsy or a genetic epilepsy syndrome;
- wherein the subject is a human;
- and
- wherein administration of Compound 1, or a pharmaceutically acceptable salt thereof, results in a reduction in the severity, number and/or frequency of seizures experienced by the subject as compared to the severity, number and/or frequency of seizures experienced by the subject prior to administration of Compound 1, or a pharmaceutically acceptable salt thereof.
A method of reducing severity, number and/or frequency of seizures in a subject in need thereof, said method comprising administering to said subject Compound 1 of the following formula:
- or a pharmaceutically acceptable salt thereof, at a dose of about 0.1 mg/kg to about 10 mg/kg, administered once daily;
- wherein the subject has a genetic epilepsy or a genetic epilepsy syndrome;
- and
- wherein the subject is a human.
Dependencies are inferred from the claim text.