Ganaxolone - PubMed (original) (raw)
Review
Ganaxolone
Virinder Nohria et al. Neurotherapeutics. 2007 Jan.
Abstract
Ganaxolone (3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one) (GNX) is the 3beta-methylated synthetic analog of allopregnanolone; it belongs to a class of compounds referred to as neurosteroids. GNX is an allosteric modulator of GABA(A) receptors acting through binding sites which are distinct from the benzodiazepine binding site. It has activity in a broad range of animal models of epilepsy. GNX has been shown to be well tolerated in adults and children. In early phase II studies, GNX has been shown to have activity in adult patients with partial-onset seizures and epileptic children with history of infantile spasms. It is currently undergoing further development in infants with newly diagnosed infantile spasms, in women with catamenial epilepsy, and in adults with refractory partial-onset seizures.
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References
- Rogawski MA, Reddy DS. Neurosteroids: endogenous modulators of seizure susceptibility. In: Rho JM, Sankar R, Cavazos JE, editors. Epilepsy: scientific foundations of clinical practice. New York: Marcel Dekker; 2004. pp. 319–355.
- Carter RB, Wood PL, Wieland S, et al. Characterization of the anticonvulsant properties of ganaxolone (CCD 1042; 3α-hydroxy-3β-methyl-5α-pregnan-20-one), a selective, high-affinity, steroid modulator of the γ-aminobutyric acidA receptor. J Pharmacol Exp Ther. 1997;280:1284–1295. - PubMed
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