Ganaxolone

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Ganaxolone

Ganaxolone (pronounced: gah-nax-oh-lone) is a synthetic neuroactive steroid and a derivative of the naturally occurring neurosteroid allopregnanolone. It was developed by Marinus Pharmaceuticals and is used in the treatment of epilepsy and post-traumatic stress disorder (PTSD).

Etymology

The term "Ganaxolone" is a combination of parts of the words "GABA" and "allopregnanolone". "GABA" refers to gamma-aminobutyric acid, the chief inhibitory neurotransmitter in the mammalian central nervous system. "Allopregnanolone" is a naturally occurring neurosteroid, from which Ganaxolone is derived.

Mechanism of Action

Ganaxolone acts on the GABA_A receptor, the major inhibitory neurotransmitter receptor in the brain. It enhances the inhibitory effects of GABA, thereby reducing neuronal excitability. This mechanism is beneficial in conditions like epilepsy, where there is excessive neuronal activity.

Related Terms

  • Neurosteroid: Steroids synthesized in the brain that are involved in a wide range of functions, including cognitive and stress response.
  • Allopregnanolone: A naturally occurring neurosteroid that Ganaxolone is derived from.
  • GABA_A receptor: A type of protein which, when bound to by certain substances, can inhibit or reduce the activity of neurons.
  • Epilepsy: A neurological disorder marked by sudden recurrent episodes of sensory disturbance, loss of consciousness, or convulsions, associated with abnormal electrical activity in the brain.
  • Post-traumatic stress disorder (PTSD): A disorder characterized by failure to recover after experiencing or witnessing a terrifying event.

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