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Current Medicinal Chemistry

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ISSN (Print): 0929-8673
ISSN (Online): 1875-533X

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Perspective

Ganaxolone: First FDA-approved Medicine for the Treatment of Seizures Associated with Cyclin-dependent Kinase-like 5 Deficiency Disorder

Author(s): Surya K. De*

Volume 31, Issue 4, 2024

Published on: 08 May, 2023

Page: [388 - 392] Pages: 5

DOI: 10.2174/0929867330666230320123952

Abstract

The neurosteroids progesterone and allopregnanolone control numerous neuroprotective functions in neural tissues, including inhibition of epileptic seizures and cell death. Ganaxolone (3α-hydroxy-3β-methyl-5α-pregnan-20-one) (GNX) is the 3β- methylated synthetic analog of allopregnanolone and an allosteric GABAA positive modulator. Ganaxolone reduces the frequency of CDD-associated seizures.

Keywords: Anti-seizure medication, ganaxolone, neurosteroids, γ-aminobutyric acid, central nervous system, clinical trial, epilepsy.

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