Ganaxolone References

Ganaxolone in Seizure Disorders

  1. Belelli et al., 2006. Neuroactive steroids and inhibitory transmission: Mechanisms of action and physiological relevance. Neuroscience 138: 821–829.
  2. Belelli et al., 2009. Extrasynaptic GABAA Receptors: Form, Pharmacology, and Function. J. Neuroscience, 29(41):12757–12763.
  3. Biagini, G., et al. 2006. Endogenous neurosteroids modulate epileptogenesis in a model of temporal lobe epilepsy. Exp Neurol 201, 519-524
  4. Biagini, G., et al., 2009. Neurosteroids and epileptogenesis in the pilocarpine model: Evidence for a relationship between P450scc induction and length of the latent period. Epilepsia 50(Suppl. 1), 53-58.
  5. Bialer M., et al., 2013. Progress report on new antiepileptic drugs: A summary of the Eleventh Eilat Conference (EILAT XI). Epilepsy Res, 103, 2-30.
  6. Carter, R.B., et al., 1997. 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 gamma-aminobutyric acidA receptor. J Pharmacol Exp Ther 280, 1284-1295.
  7. Herd, M.B., et al., 2007. Neurosteroid modulation of synaptic and extrasynaptic GABAA receptors. Pharmacol Ther 116, 20-34.
  8. Hosie, A.D., et al., 2006. Endogenous neurosteroids regulate GABAA receptors through two discrete transmembrane sites. Nature 444, 486-489
  9. Kaminski, R. M., et al., 2003.Protective efficacy of neuroactive steroids against cocaine kindled-seizures in mice. Eur J Pharmacol 474, 217-22.
  10. Kaminski, R.M., et al., 2004. Allopregnanolone analogs that positively modulate GABAA receptors protect against partial seizures induced by 6-Hz electrical stimulation in mice. Epilepsia 45, 864-867.
  11. Kerrigan, J. F., et al., 2000. Ganaxolone for treating intractable infantile spasms: a multicenter, open-label, add-on trial. Epilepsy Res 42, 133-139.
  12. Lawrence et al. 2010. Endogenous neurosteroid synthesis modulates seizure frequency. Ann Neurol. 67(5):689-93.
  13. Laxer et al., 2000. Assessment of Ganaxolone’s Anticonvulsant Activity Using a Randomized, Double-Blind, Presurgical Trial Design. Epilepsia 41(9):1187-94.
  14. Lemos, T., Cavalheiro, E.A., 1995. Suppression of pilocarpine-induced status epilepticus and the late development of epilepsy in rats. Exp Brain Res 102, 423-428.
  15. Leśkiewicz, M., et al., 2003. Inhibitory effect of some neuroactive steroids on cocaine-induced kindling in mice. Pol J Pharmacol 55, 1131-1136.
  16. Liptáková, S., et al., 2000. Effect of ganaxolone on flurothyl seizures in developing rats. Epilepsia 41, 788-793.
  17. Nohria, V., Giller, E., 2007. Ganaxolone. Neurotherapeutics 4, 102-105.
  18. Pieribone, V.A., et al., 2007. Clinical evaluation of ganaxolone in pediatric and adolescent patients with refractory epilepsy. Epilepsia 48, 1870-1874.
  19. Reddy, D.S., Rogawski, M.A., 2000. Chronic treatment with the neuroactive steroid ganaxolone in the rat induces anticonvulsant tolerance to diazepam but not to itself. J Pharmacol Exp Ther 295, 1241-1248.
  20. Reddy, D.S., Rogawski, M.A., 2010. Ganaxolone suppression of behavioral and electrographic seizures in the mouse amygdala kindling model. Epilepsy Res doi:10.1016/j.eplepsyres.2010.01.009.
  21. Reddy and Rogawski 2012. Neurosteroids — Endogenous Regulators of Seizure Susceptibility and Role in the Treatment of Epilepsy. In: Jasper’s Basic Mechanisms of the Epilepsies 4th ed. Bethesda (MD): National Center for Biotechnology Information (US).

Neurosteroids in Fragile X Syndrome

  1. Paluszkiewicz et al., (2011) Fragile X Syndrome: The GABAergic System and Circuit Dysfunction, Devel. Neurosci., 33, 349-364.
  2. Gantois, Vandesompele et al., (2006) Expression profiling suggests underexpression of the GABAA receptor subunit y in the fragile X knockout mouse model, Neurobio. Dis. 21:346 – 357.
  3. Huelens, D’Hulst et al., (2012) Pharmacological treatment of fragile X syndrome with GABAergic drugs in a knockout mouse model, Behav. Brain Res. 229:244–24.

PCDH19 Pediatric Epilepsy

  1. Gecz et al., 2015. Mutations of protocadherin 19 in female epilepsy (PCDH19-FE) lead to allopregnanolone deficiency. Human Molecular Genetics. ddv245v1-ddv245.