Ganaxolone
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Ganaxolone is a neuroactive steroid GABA-A receptor modulator used for the treatment of seizures associated with CDKL5 deficiency disorder (CDD).
Ganaxolone is a neuroactive steroid GABA-A receptor modulator used for the treatment of seizures associated with CDKL5 deficiency disorder (CDD).
Ganaxolone belongs to a novel class of neuroactive steroids sometimes referred to as "epalons", which are potent and specific positive allosteric modulators of γ-aminobutyric acid type A (GABA) receptors in the central nervous system (CNS). It binds GABA at one of several potential binding sites, all of which are distinct from the benzodiazepine binding site. By enhancing the inhibitory effects of GABA receptors, endogenous and exogenous neurosteroids have been associated with anxiolytic, sedative, and anticonvulsant effects, amongst others. While the precise mechanism of action of ganaxolone in the treatment of seizures associated with CDD is unknown, its anticonvulsant effects are likely due to positive allosteric GABA modulation
Ganaxolone is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients ≥2 years old by the FDA. It is also approved as an adjuvant treatment for the same condition in patients aged 2 to 17, although it may be continued in patients 18 years old or older, by the EMA.
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