AR122744A1 - COMPOUNDS AND METHODS FOR REDUCING THE EXPRESSION OF KCNT1 - Google Patents

COMPOUNDS AND METHODS FOR REDUCING THE EXPRESSION OF KCNT1

Info

Publication number
AR122744A1
AR122744A1 ARP210101756A ARP210101756A AR122744A1 AR 122744 A1 AR122744 A1 AR 122744A1 AR P210101756 A ARP210101756 A AR P210101756A AR P210101756 A ARP210101756 A AR P210101756A AR 122744 A1 AR122744 A1 AR 122744A1
Authority
AR
Argentina
Prior art keywords
compounds
methods
kcnt1
reducing
pharmaceutical compositions
Prior art date
Application number
ARP210101756A
Other languages
Spanish (es)
Inventor
Susan M Freier
Paymaan Jafar-Nejad
Huynh-Hoa Bui
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of AR122744A1 publication Critical patent/AR122744A1/en

Links

Abstract

Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o actividad del ARN de KCNT1 en una célula o sujeto, y en determinadas circunstancias reducir la cantidad de proteína de KCNT1 en una célula o un sujeto. Estos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o característica de una afección neurológica. Dichos síntomas y características incluyen convulsiones, encefalopatía y anomalías del comportamiento. Los ejemplos no taxativos de afecciones neurológicas que se benefician de estos compuestos, métodos y composiciones farmacéuticas son la epilepsia de la infancia con crisis focales migratorias (EIMFS), la epilepsia del lóbulo frontal nocturna autosómica dominante (ADNFLE), el síndrome de West y el síndrome de Ohtahara.Compounds, methods, and pharmaceutical compositions are provided for reducing the amount or activity of KCNT1 RNA in a cell or subject, and in certain circumstances reducing the amount of KCNT1 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful for ameliorating at least one symptom or characteristic of a neurological condition. Such symptoms and features include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological conditions that benefit from these compounds, methods and pharmaceutical compositions are epilepsy of childhood with migratory focal seizures (EIMFS), autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE), West syndrome and Ohtahara syndrome.

ARP210101756A 2020-06-25 2021-06-25 COMPOUNDS AND METHODS FOR REDUCING THE EXPRESSION OF KCNT1 AR122744A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063044345P 2020-06-25 2020-06-25

Publications (1)

Publication Number Publication Date
AR122744A1 true AR122744A1 (en) 2022-10-05

Family

ID=83835067

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210101756A AR122744A1 (en) 2020-06-25 2021-06-25 COMPOUNDS AND METHODS FOR REDUCING THE EXPRESSION OF KCNT1

Country Status (1)

Country Link
AR (1) AR122744A1 (en)

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