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

Ganaxolone in Status Epilepticus

  1. Saporito MS, Gruner, JA, et al. Intravenously administered ganaxolone blocks diazepam-resistant lithium-pilocarpine-induced status epilepticus in rats: Comparison with allopregnanolone. J Pharmacology Exp Ther. March 2019. 368: 326-337. Doi:10.1124/jpet.118.252155
  2. 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 American Epilepsy Society Annual Meeting. December 2018.
  3. Ramsay RE, Husain AM, et al. Pharmacokinetic and pharmacodynamic (PK/PD) relationship of intravenous ganaxolone in refractory status epilepticus. Poster presentation at the American Epilepsy Society Annual Meeting. December 2019.
  4. Husain AM, Wu, H et al. Population pharmacokinetic/pharmacodynamic modeling of the electroencephalographic effects of ganaxolone in healthy subjects. Poster presentation at the American Epilepsy Society Annual Meeting. December 2019.

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.
  5. Specchio N, Masuoka L, et al. Long-term, durable seizure frequency reduction in children with CDKL5 deficiency disorder (CDD) treated with ganaxolone. Poster presentation at the American Epilepsy Society annual meeting. December 2018.

Background on PCDH19 Related Epilepsy

  1. Sullivan J, Specchio N, et al. Preliminary evidence of a predictive clinical biomarker in PCDH19-related epilepsy: significant treatment effect of ganaxolone in biomarker-positive patients. Poster presentation at American Epilepsy Society annual meeting. December 2018.
  2. 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
  3. 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
  4. 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.

Ganaxolone in Postpartum Depression

  1. Maximos B, Riesenberg R. Safety and efficacy of intravenous ganaxolone in severe postpartum depression: results from a double-blind, placebo-controlled phase 2 study. Poster presentation at annual meeting of Society of Biological Psychiatry. 2019.
  2. Maguire J, Mody I. Steroid hormone fluctuations and GABAAR plasticity. Psychoneuroendocrinology. 2009;34(SUPPL. 1). doi:10.1016/j.psyneuen.2009.06.019
  3. 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

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

Ganaxolone in Focal Onset Seizures

  1. Lappalainen J, Tsai J, et al. A multicenter, double-blind, randomized, placebo-controlled phase 3 trial to determine the efficacy and safety of ganaxolone as adjunctive therapy for adults with drug-resistant focal-onset seizures. Poster presentation at the 69th Annual Meeting American Academy of Neurology. 2017.
  2. Authored by Marinus employees. The pharmacokinetics of two oral ganaxolone formulations under fed and fasted conditions.  Poster presentation at the American Society of Epilepsy Annual Meeting.  December 2018.


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