Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Experiment Videos

Ganaxolone.

Virinder Nohria1, Earl Giller

  • 1Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia, USA. vnohria@aol.com

Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics
|January 3, 2007
PubMed
Summary
This summary is machine-generated.

Related Concept Videos

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Immune Thrombocytopenia.

The New England journal of medicine·2020
Same author

Comparison of Site-Based Versus Central Ratings in a Study of Generalized Anxiety Disorder.

Journal of clinical psychopharmacology·2015
Same author

A novel design for a dose finding, safety, and drug interaction study of an antiepileptic drug (retigabine) in early clinical development.

International journal of clinical pharmacology and therapeutics·2014
Same author

Adjunctive use of ezogabine/retigabine with either traditional sodium channel blocking antiepileptic drugs (AEDs) or AEDs with other mechanisms of action: Evaluation of efficacy and tolerability.

Epilepsy research·2014
Same author

A 6-week randomized, double-blind, placebo-controlled, comparator referenced trial of vabicaserin in acute schizophrenia.

Journal of psychiatric research·2014
Same author

Safety and tolerability of different titration rates of retigabine (ezogabine) in patients with partial-onset seizures.

Epilepsy research·2013
Same journal

Sotagliflozin improves cognitive deficits and attenuates neuroinflammation of Alzheimer's disease.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2026
Same journal

Neurological complications of current and emerging CAR-T cell therapies.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2026
Same journal

Hemodynamics Beyond Ischemic Zone Underlies Early Neurological Deterioration in Minor Stroke with Large Vessel Occlusion.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2026
Same journal

Nervonic acid and the long arc of therapeutic hope in X-linked adrenoleukodystrophy.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2026
Same journal

Long-term efficacy of subthalamic nucleus deep brain stimulation in focal motor seizures and implications for candidate selection.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2026
Same journal

Brain interstitial fluid pharmacokinetics and therapeutic effect of a BBB penetrating amyloid beta antibody measured by microdialysis.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2026
See all related articles

Ganaxolone (GNX), a neurosteroid, modulates GABA(A) receptors and shows promise in treating various epilepsy types. Clinical studies indicate good tolerability and efficacy in adults and children with specific seizure disorders.

Area of Science:

  • Neuroscience
  • Pharmacology

Background:

  • Ganaxolone (GNX) is a synthetic neurosteroid and a 3beta-methylated analog of allopregnanolone.
  • It functions as an allosteric modulator of GABA(A) receptors at unique binding sites.

Purpose of the Study:

  • To evaluate the efficacy and tolerability of Ganaxolone (GNX) in various epilepsy models and patient populations.
  • To explore GNX's potential in treating partial-onset seizures, infantile spasms, catamenial epilepsy, and refractory epilepsy.

Main Methods:

  • Preclinical studies in animal models of epilepsy.
  • Phase II clinical trials in adult patients with partial-onset seizures.
  • Clinical studies in epileptic children with a history of infantile spasms.

Main Results:

Related Experiment Videos

  • Ganaxolone demonstrated activity across a broad range of animal epilepsy models.
  • Early phase II studies showed efficacy in adult partial-onset seizures and pediatric infantile spasms.
  • GNX was well tolerated in both adult and pediatric populations.

Conclusions:

  • Ganaxolone is a well-tolerated neurosteroid with demonstrated efficacy in specific epilepsy types.
  • Ongoing trials are investigating GNX for infantile spasms, catamenial epilepsy, and refractory partial-onset seizures.