Ganaxolone, an approved therapy for CDKL5-Deficiency Disorder, is an inhibitor of PTP1B - PubMed
4 hours ago
- #CDKL5-Deficiency Disorder
- #Ganaxolone
- #PTP1B inhibitor
- Ganaxolone is an FDA-approved therapy for CDKL5-Deficiency Disorder (CDD) used to treat seizures in patients aged 2 years and older.
- The study reveals that ganaxolone acts as a PTP1B inhibitor, similar in potency to known inhibitors like MSI-1436, in both in vitro and cellular models.
- Inhibition of PTP1B in CDKL5-KO cells enhances TRKB/BDNF signaling, restores cellular morphology, and suggests targeting PTP1B as a therapeutic strategy for CDD.