Nohria Virinder, Giller Earl
Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA.
Neurotherapeutics. 2007 Jan;4(1):102-5. doi: 10.1016/j.nurt.2006.11.003.
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.
加奈索酮（3α-羟基-3β-甲基-5α-孕烷-20-酮）（GNX）是别孕烷醇酮的3β-甲基化合成类似物；它属于一类被称为神经甾体的化合物。GNX是GABA(A)受体的变构调节剂，通过与不同于苯二氮䓬结合位点的结合位点起作用。它在广泛的癫痫动物模型中具有活性。已证明GNX在成人和儿童中耐受性良好。在II期早期研究中，已证明GNX对部分性发作的成年患者和有婴儿痉挛病史的癫痫儿童有活性。目前，它正在新诊断为婴儿痉挛的婴儿、月经性癫痫的女性以及难治性部分性发作的成人中进行进一步开发。
