Find references to ganaxolone in the following research literature.


Overview of Neurosteroids in Various CNS Indications

  1. MacKenzie G, Maguire J. Neurosteroids and GABAergic signaling in health and disease. Biomol Concepts. 2013;4(1):29-42. doi:10.1515/bmc-2012-0033
  2. Zorumski CF, Paul SM, Izumi Y, Covey DF, Mennerick S. Neurosteroids, stress and depression: Potential therapeutic opportunities. Neurosci Biobehav Rev. 2013;37(1):109-122. doi:10.1016/j.neubiorev.2012.10.005
  3. Reddy DS. Role of hormones and neurosteroids in epileptogenesis. Front Cell Neurosci. 2013;7(July):1-20. doi:10.3389/fncel.2013.00115

Ganaxolone Mechanism of Action

  1. 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(3):1284-1295.
  2. Nik AM, Pressly B, Singh V, et al. Rapid Throughput Analysis of GABA A Receptor Subtype Modulators and Blockers Using DiSBAC 1 (3) Membrane Potential Red Dye. Mol Pharmacol. 2017;92(1):88-99. doi:10.1124/mol.117.108563.
  3. Chuang S-H, Reddy DS. 3β-Methyl-Neurosteroid Analogs are Preferential Positive Allosteric Modulators and Direct Activators of Extrasynaptic δGABA-A Receptors in the Hippocampus Dentate Gyrus Subfield. J Pharmacol Exp Ther. 2018;246660:jpet.117.246660. doi:10.1124/jpet.117.246660

Background on CDKL5 Deficiency Disorder

  1. Fehr S, Wong K, Chin R, et al. Seizure variables and their relationship to genotype and functional abilities in the CDKL5 disorder. Neurology. 2016;87(21):2206-2213. doi:10.1212/WNL.0000000000003352
  2. Mangatt M, Wong K, Anderson B, et al. Prevalence and onset of comorbidities in the CDKL5 disorder differ from Rett syndrome. Orphanet J Rare Dis. 2016;11(1). doi:10.1186/s13023-016-0418-y
  3. Müller A, Helbig I, Jansen C, et al. Retrospective evaluation of low long-term efficacy of antiepileptic drugs and ketogenic diet in 39 patients with CDKL5-related epilepsy. Eur J Paediatr Neurol. 2016;20(1):147-151. doi:10.1016/j.ejpn.2015.09.001
  4. Specchio N, Chez M, Tarquinio D, et al. Ganaxolone in children with CDKL5 gene-related epileptic encephalopathy: top-line data from an open-label trial. Poster presentation at the Child Neurology Society 46th Annual Meeting. October 2017.

Background on PCDH19 Related Epilepsy

  1. Tan C, Shard C, Ranieri E, et al. Mutations of protocadherin 19 in female epilepsy (PCDH19-FE) lead to allopregnanolone deficiency. Hum Mol Genet. 2015;24(18):5250-5259. doi:10.1093/hmg/ddv245
  2. Smith L, Singhal N, El Achkar CM, et al. PCDH19-related epilepsy is associated with a broad neurodevelopmental spectrum. Epilepsia. 2018;59(3):679-689. doi:10.1111/epi.14003
  3. Lappalainen J, Chez M, Sullivan J, et al. A multi-center, open-label trial of ganaxolone in children with PCDH19 epilepsy. Poster presentation at the American Academy of Neurology 69th Annual Meeting. April 2017.

Rationale for Ganaxolone in Postpartum Depression

  1. Maguire J, Mody I. Steroid hormone fluctuations and GABAAR plasticity. Psychoneuroendocrinology. 2009;34(SUPPL. 1). doi:10.1016/j.psyneuen.2009.06.019
  2. Evans SEG, Ross LE, Sellers EM, Purdy RH, Romach MK. 3Α-Reduced Neuroactive Steroids and Their Precursors During Pregnancy and the Postpartum Period. Gynecol Endocrinol. 2005;21(5):268-279. doi:10.1080/09513590500361747

Rationale for Ganaxolone in Fragile X

  1. Ligsay A, Van Dijck A, Nguyen D V., et al. A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome. J Neurodev Disord. 2017;9(1). doi:10.1186/s11689-017-9207-8

Rationale for Ganaxolone in Status Epilepticus

  1. Saporito MS, Henry D, Barker-Heliski ML, et al. Ganaxolone administered intravenously prevents behavioral seizures promotes survival in the rat lithium-pilocarpine model of status epilepticus. Poster presentation at American Academy of Neurology 68th Annual Meeting. April 2016.
  2. Saporito MS, Gruner JA, Tsai J, et al. Ganaxolone and diazepam administered IV produce a synergistic antiepileptic effect in a treatment refractory model of status epilepticus. Poster presentation at the American Epilepsy Society 70th Annual Meeting. December 2016.
  3. Tsai J, Guptill J, MacLeod D, et al. Phase 1 study to determine the pharmacokinetics, pharmacodynamics, and safety of IV ganaxolone in healthy adults. Poster presentation at the 7th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures. April 2017.


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