AR121445A1 - COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION - Google Patents
COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSIONInfo
- Publication number
- AR121445A1 AR121445A1 ARP210100503A ARP210100503A AR121445A1 AR 121445 A1 AR121445 A1 AR 121445A1 AR P210100503 A ARP210100503 A AR P210100503A AR P210100503 A ARP210100503 A AR P210100503A AR 121445 A1 AR121445 A1 AR 121445A1
- Authority
- AR
- Argentina
- Prior art keywords
- dysfunctions
- compounds
- methods
- modulating
- pharmaceutical compositions
- Prior art date
Links
Landscapes
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para modular la proteína y/o ARN de SCN1A en una célula o sujeto. Tales compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma de una enfermedad encefalopática epiléptica o del desarrollo, tal como el síndrome de Dravet. Dichos síntomas incluyen convulsiones, muerte súbita inesperada en la epilepsia, estado epiléptico, retrasos en el comportamiento y el desarrollo, disfunciones del movimiento y el equilibrio, afecciones ortopédicas, disfunciones motoras y cognitivas, problemas de retraso en el lenguaje y el habla, disfunciones de integración visomotora, disfunciones de percepción visual, disfunciones ejecutivas, problemas de crecimiento y nutrición, dificultades para dormir, infecciones crónicas, trastornos de integración sensorial y disautonomía.Compounds, methods, and pharmaceutical compositions for modulating SCN1A protein and/or RNA in a cell or subject are provided. Such compounds, methods and pharmaceutical compositions are useful for ameliorating at least one symptom of an epileptic or developmental encephalopathic disease, such as Dravet syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral and developmental delays, movement and balance dysfunctions, orthopedic conditions, motor and cognitive dysfunctions, delayed speech and language problems, visuomotor integration, visual perception dysfunctions, executive dysfunctions, growth and nutrition problems, sleeping difficulties, chronic infections, sensory integration disorders and dysautonomia.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062983555P | 2020-02-28 | 2020-02-28 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR121445A1 true AR121445A1 (en) | 2022-06-08 |
Family
ID=82401565
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP210100503A AR121445A1 (en) | 2020-02-28 | 2021-02-26 | COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION |
Country Status (1)
| Country | Link |
|---|---|
| AR (1) | AR121445A1 (en) |
-
2021
- 2021-02-26 AR ARP210100503A patent/AR121445A1/en unknown
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