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GABA receptor allosteric modulator receives priority review qualification, significantly reduces seizure frequency

 Last Update: 2021-09-28
 Source: Internet
 Author: User

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fragment based drug discovery

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Recently, Marinus Pharmaceuticals announced that the U.

This new drug application is based on data support from a phase 3 clinical trial, and a total of 101 patients were enrolled

In terms of safety, ganaxolone was generally well tolerated in this phase 3 trial and showed safety characteristics consistent with previous clinical trials.

CDKL5 deficiency is a serious and rare genetic disease caused by mutations in the cyclin-dependent kinase-like 5 (CDKL5) gene located on the X chromosome

Ganaxolone is an under-researched gender modulator targeting GABAA receptors.

Reference materials:

[1] Marinus Pharmaceuticals Announces FDA Acceptance for Filing and Priority Review of New Drug Application for Ganaxolone in CDKL5 Deficiency Disorder.

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