Article

Ganaxolone, an approved therapy for CDKL5-Deficiency Disorder, is an inhibitor of PTP1B

2025-11-01

Abstract excerpt

CDKL5-deficiency disorder or CDD, which results in intellectual disability, speech and motor deficits, and seizures that can start as early as six weeks after birth, is caused by de novo mutations in the CDKL5 gene. In early 2022, the FDA approved Ganaxolone (Commercial name: Ztalmy) for treatment of seizures in CDD patients 2 years and older. Ganaxolone has been reported to act as a GABAA receptor agonist that h...

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Literature Corpus work: 5daf3b62-78ba-5765-a7d5-502ef900210b
DOI: 10.1101/2025.10.31.685887

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