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Current Medicinal Chemistry

Author(s): Surya K. De*

DOI: 10.2174/0929867330666230320123952

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

Page: [388 - 392] Pages: 5

  • * (Excluding Mailing and Handling)

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.

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