ArticleThe Journal of biological chemistry2026
Ganaxolone, an approved therapy for CDKL5-deficiency disorder, is an inhibitor of PTP1B.
Article in The Journal of biological chemistry, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
CDKL5-deficiency disorder or CDD, which results in intellectual disability, speech, and motor deficits, and seizures that can start as early as 6 weeks after birth, is caused by de novo mutations in the CDKL5 gene. In early 2022, the FDA approved ganaxolone (Commercial name: Ztalmy) for the treatment of seizures in CDD patients 2 years and older. Ganaxolone has been reported to act as a GABA
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